On January 25, 2022, Nature published an article listing seven technologies worthy of attention this year. Targeted genetic therapies was on the list. The remaining six technologies are: Fully finished genomes, Protein structure solutions, Quantum simulation, Precise genome manipulation, Spatial multi-omics), CRISPR-based diagnostics.
Gene therapy involves techniques that modify or manipulate genes to treat or prevent diseases. The first gene therapy treatment occurred in 1990 for severe combined immunodeficiency. There are four main approaches to gene therapy: inserting a normal gene to compensate for a defective one, replacing an abnormal gene with a normal one, repairing an abnormal gene, or altering gene regulation. Viruses are commonly used as vectors to deliver therapeutic genes into target cells, with retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses being some of the most widely used viral vectors, each with advantages and limitations.
Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or at least to improve the clinical status of a patient.
One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells.
Safe methods have been devised to do this, using several viral and non-viral vectors.
In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery.
The biggest hurdle faced by medical research in gene therapy is the availability of effective gene-carrying vectors that meet all of the following criteria:
Protection of transgene or genetic cargo from degradative action of systemic and endonucleases,
Delivery of genetic material to the target site, i.e., either cell cytoplasm or nucleus,
Low potential of triggering unwanted immune responses or genotoxicity,
Economical and feasible availability for patients .
Viruses are naturally evolved vehicles that efficiently transfer their genes into host cells.
Choice of viral vector is dependent on gene transfer efficiency, capacity to carry foreign genes, toxicity, stability, immune responses towards viral antigens and potential viral recombination.
There are a wide variety of vectors used to deliver DNA or oligo nucleotides into mammalian cells, either in vitro or in vivo.
The most common vector system based on retroviruses, adenoviruses, herpes simplex viruses, adeno associated viruses.
This document discusses adenoviral cloning vectors. It begins by defining a cloning vector as a small piece of DNA that can be stably maintained in an organism and have foreign DNA inserted into it for cloning purposes. It then discusses viral vectors, noting that they are commonly used to deliver genetic material into cells through transduction. The document focuses on properties of viral vectors, specifically safety features and targeting abilities. It provides details on adenoviruses, noting they can efficiently transfer genes, their structure, applications in gene therapy and vaccination, and their DNA genome capacity. Adeno-associated viruses are also mentioned as attractive for gene therapy due to mild immune response.
The Recent advances in gene delivery using nanostructures and future prospectsAANBTJournal
This document summarizes recent advances in using nanostructures for gene delivery in gene therapy. It discusses the key challenges to effective gene therapy, including overcoming intracellular and extracellular barriers to delivery. It reviews the history of viral and non-viral gene delivery methods. Specifically, it describes several non-viral methods that have been developed using nanostructures, such as magnetic nanoparticles, PEGylated multi-component carriers, oligonucleotides, lipoplexes, polyplexes, and dendrimers. Overall, it finds that while viral methods remain more effective, recent advances in non-viral nanostructure-based delivery systems show promise for improving safety and effectiveness of gene therapy.
This document discusses gene transfer techniques, including viral and non-viral delivery systems. It describes gene therapy as using genes to treat disease by inserting a gene into a patient's cells instead of using drugs or surgery. It outlines various non-viral physical methods like gene guns, ultrasound, electroporation, and magnetofection and chemical methods like cationic liposomes and polymers to facilitate gene transfer. Viral vectors discussed include retroviruses, adenoviruses, and adeno-associated viruses. In conclusion, while progress has been made, developing safe and effective non-viral delivery systems for in vivo gene therapy remains a challenge.
Gene therapy involves techniques that modify or manipulate genes to treat or prevent diseases. The first gene therapy treatment occurred in 1990 for severe combined immunodeficiency. There are four main approaches to gene therapy: inserting a normal gene to compensate for a defective one, replacing an abnormal gene with a normal one, repairing an abnormal gene, or altering gene regulation. Viruses are commonly used as vectors to deliver therapeutic genes into target cells, with retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses being some of the most widely used viral vectors, each with advantages and limitations.
Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or at least to improve the clinical status of a patient.
One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells.
Safe methods have been devised to do this, using several viral and non-viral vectors.
In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery.
The biggest hurdle faced by medical research in gene therapy is the availability of effective gene-carrying vectors that meet all of the following criteria:
Protection of transgene or genetic cargo from degradative action of systemic and endonucleases,
Delivery of genetic material to the target site, i.e., either cell cytoplasm or nucleus,
Low potential of triggering unwanted immune responses or genotoxicity,
Economical and feasible availability for patients .
Viruses are naturally evolved vehicles that efficiently transfer their genes into host cells.
Choice of viral vector is dependent on gene transfer efficiency, capacity to carry foreign genes, toxicity, stability, immune responses towards viral antigens and potential viral recombination.
There are a wide variety of vectors used to deliver DNA or oligo nucleotides into mammalian cells, either in vitro or in vivo.
The most common vector system based on retroviruses, adenoviruses, herpes simplex viruses, adeno associated viruses.
This document discusses adenoviral cloning vectors. It begins by defining a cloning vector as a small piece of DNA that can be stably maintained in an organism and have foreign DNA inserted into it for cloning purposes. It then discusses viral vectors, noting that they are commonly used to deliver genetic material into cells through transduction. The document focuses on properties of viral vectors, specifically safety features and targeting abilities. It provides details on adenoviruses, noting they can efficiently transfer genes, their structure, applications in gene therapy and vaccination, and their DNA genome capacity. Adeno-associated viruses are also mentioned as attractive for gene therapy due to mild immune response.
The Recent advances in gene delivery using nanostructures and future prospectsAANBTJournal
This document summarizes recent advances in using nanostructures for gene delivery in gene therapy. It discusses the key challenges to effective gene therapy, including overcoming intracellular and extracellular barriers to delivery. It reviews the history of viral and non-viral gene delivery methods. Specifically, it describes several non-viral methods that have been developed using nanostructures, such as magnetic nanoparticles, PEGylated multi-component carriers, oligonucleotides, lipoplexes, polyplexes, and dendrimers. Overall, it finds that while viral methods remain more effective, recent advances in non-viral nanostructure-based delivery systems show promise for improving safety and effectiveness of gene therapy.
This document discusses gene transfer techniques, including viral and non-viral delivery systems. It describes gene therapy as using genes to treat disease by inserting a gene into a patient's cells instead of using drugs or surgery. It outlines various non-viral physical methods like gene guns, ultrasound, electroporation, and magnetofection and chemical methods like cationic liposomes and polymers to facilitate gene transfer. Viral vectors discussed include retroviruses, adenoviruses, and adeno-associated viruses. In conclusion, while progress has been made, developing safe and effective non-viral delivery systems for in vivo gene therapy remains a challenge.
The concept of transferring genes to tissues for clinical applications has been discussed for nearly half a century, but the ability to manipulate genetic material via recombinant DNA technology has brought this goal to reality. ‘Gene Therapy’ covers both the research and clinical applications of the new genetic therapy techniques currently being developed. The application of molecular biology has revolutionized researchers understanding of many diseases and has been readily applied for diagnostic purposes. Now-a-day this is originally conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, gene therapy now is considered for many non–life-threatening conditions, including those adversely affecting a patient’s quality of life. The lack of suitable treatment has become a rational basis for extending the scope of gene therapy. It is not very far, the justifiable optimism that with increased biotechnological improvement, gene therapy will become a standard part of clinical practice.
This document provides an overview of gene therapy and various gene transfer techniques. It discusses that gene therapy uses genes to treat or prevent disease by inserting genes into patient's cells. There are two main types of gene transfer techniques - non-viral and viral delivery systems. Non-viral techniques include physical methods like gene guns and electroporation, as well as chemical methods like cationic liposomes and polymers. Viral vectors are commonly used due to their efficiency and include retroviruses, adenoviruses, and adeno-associated viruses. The document reviews several applications of these various gene transfer techniques and concludes that while progress has been made, more development is still needed to design a safe and effective delivery system that can be
This document provides an overview of gene therapy. It discusses the basic concept of replacing a mutated gene with a healthy copy to cure diseases. It describes the central dogma of molecular biology and barriers to gene therapy like developing effective carriers or vectors. The main types of vectors discussed are viral vectors, including RNA virus vectors like retroviruses and DNA virus vectors like adenoviruses and adeno-associated viruses. Clinical trials of gene therapy are also mentioned, with the first using retroviruses to treat severe combined immunodeficiency. In conclusion, the document states that gene therapy requires identifying target genes, developing safe and efficient vectors, and conducting clinical trials optimized for specific diseases.
DNA lipofection - Efficiency in invitro and invivo transfectionpugazhenthi6
This document discusses DNA lipofection and its efficiency in in vitro and in vivo transfection. It begins by introducing gene therapy and its goal of introducing genes to prevent or cure diseases. It then describes the two main types of gene therapy: somatic cell gene therapy, which does not affect future generations; and germ line gene therapy, which is heritable but not currently attempted. The document outlines approaches to gene therapy, including ex vivo and in vivo methods. It discusses various vectors used in gene therapy such as viral vectors like retroviruses and adenoviruses and non-viral vectors like lipoplexes. The document concludes by noting both the disadvantages of potential immune responses and difficulties treating multiple gene disorders through gene therapy and
This document provides an overview of gene therapy. It defines gene therapy as using genes or oligonucleotide sequences as therapeutic molecules to treat genetic defects. The document describes the types of gene therapy, strategies used, methods of delivery including ex vivo and in vivo approaches, target cells, vectors, advantages and disadvantages. It also discusses the current status of gene therapy and diseases where successful clinical trials have been reported.
This document provides an overview of gene therapy, including what it is, different types and approaches, vectors used, methods of delivery, advantages and disadvantages. Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for a disease. It can be done via in vivo or ex vivo methods. Viral and non-viral vectors are used to deliver genes. While gene therapy holds promise to treat genetic diseases, it also faces challenges such as short-lived effects and safety issues.
This document provides an overview of gene therapy. It defines gene therapy as an experimental technique for correcting defective genes responsible for disease. It describes the main approaches like somatic cell gene therapy and germline gene therapy. It also discusses viral and non-viral vectors, delivery methods like in vivo and ex vivo, advantages like curing genetic diseases, and challenges like short-term effects and safety issues. Recent developments show promise for treating diseases like blindness and Parkinson's.
Gene therapy involves introducing genes into cells to treat disease. It works by correcting defective genes that cause diseases. The first approved gene therapy occurred in 1990 when a child with ADA-SCID was treated. There are two main types of gene therapy - somatic cell therapy targets body cells while germline therapy affects eggs and sperm. Significant challenges remain for gene therapy including developing safe methods for long-term effects and ensuring equitable access to new treatments. However, gene therapy has potential to cure many inherited diseases and revolutionize medicine.
Gene therapy is the process of inserting therapeutic genes into cells to prevent or cure wide range of diseases. The newly introduced genes will encode proteins and correct the deficiencies that occur in genetic diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease, and keep organism in good health. It is a technique for correcting defective genes responsible for disease and development.
Gene therapy involves inserting normal genes into a person's cells to treat a disease. There are several strategies for gene therapy, including ex vivo and in vivo methods. Viral vectors are commonly used to deliver therapeutic genes to target cells. However, gene therapy has faced challenges such as short-lived effects, immune responses, and safety issues with viral vectors. Continued research is working to overcome these issues and further develop promising applications of gene therapy.
Gene therapy advanced treatments for a new era aranca special reportAranca
Aranca's Report on Gene Therapy - a promising tool for Cancer, Parkinson's, HIV, severe combined immuno-deficiencies, hemophilia etc. In this report, you will discover the challenges associated with Gene Therapy as well as its expected future.
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
The document discusses gene therapy and its potential to treat genetic diseases. It describes how gene therapy works by introducing functional genes into cells to replace defective genes causing disease. The first approved gene therapy treated a girl with ADA-SCID by inserting a functional ADA gene. While promising, gene therapy faces challenges like short-lived effects and safety issues that must still be addressed.
Gene therapy involves modifying genes to treat or cure disease. It works by replacing mutated genes, inactivating abnormal genes, or introducing new genes. Early successes treated immune deficiencies, but challenges remain in achieving long-term effects without side effects. Promising areas are treating inherited retinal diseases and Parkinson's through localized delivery of therapeutic genes using viral or non-viral vectors. While offering potential cures, gene therapy also raises ethical issues that require ongoing discussion.
Emerging viral diseases pose a major threat and are becoming more common due to factors like globalization and urbanization. New technologies are helping address this issue, with genomic sequencing identifying viruses and rapid PCR diagnosis deployed in outbreak settings. Real-time PCR has been particularly useful for differentiating viral from bacterial infections during disease outbreaks. Continued development of antiviral drugs and vaccines remains an important focus, but rapid diagnostics can also help control disease spread through early case identification and contact tracing.
Cyclic Peptides Current Status & Future Prospects.pdfDoriaFang
Cyclic peptides have potential advantages over linear peptides as drug candidates due to increased stability, binding affinity, and membrane permeability. Many cyclic peptides are approved drugs or in clinical trials. Recent trends include using modern technologies to discover novel cyclic peptides and developing peptide-drug conjugates to selectively deliver therapeutic payloads. Over 50 cyclic peptides have been approved, including pegcetacoplan which targets C3 complement proteins. Hundreds of cyclic peptides are in the research pipeline, and their prospects for drug development remain promising.
Antibody–Oligonucleotide Conjugates (AOCs) in Clinical Trials.pdfDoriaFang
Summary of Antibody–Oligonucleotide Conjugates(AOCs) in Clinical Trials, including products from Avidity Biosciences, Dyne Therapeutics, Tallac Therapeutics and Denali Therapeutics.
The concept of transferring genes to tissues for clinical applications has been discussed for nearly half a century, but the ability to manipulate genetic material via recombinant DNA technology has brought this goal to reality. ‘Gene Therapy’ covers both the research and clinical applications of the new genetic therapy techniques currently being developed. The application of molecular biology has revolutionized researchers understanding of many diseases and has been readily applied for diagnostic purposes. Now-a-day this is originally conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, gene therapy now is considered for many non–life-threatening conditions, including those adversely affecting a patient’s quality of life. The lack of suitable treatment has become a rational basis for extending the scope of gene therapy. It is not very far, the justifiable optimism that with increased biotechnological improvement, gene therapy will become a standard part of clinical practice.
This document provides an overview of gene therapy and various gene transfer techniques. It discusses that gene therapy uses genes to treat or prevent disease by inserting genes into patient's cells. There are two main types of gene transfer techniques - non-viral and viral delivery systems. Non-viral techniques include physical methods like gene guns and electroporation, as well as chemical methods like cationic liposomes and polymers. Viral vectors are commonly used due to their efficiency and include retroviruses, adenoviruses, and adeno-associated viruses. The document reviews several applications of these various gene transfer techniques and concludes that while progress has been made, more development is still needed to design a safe and effective delivery system that can be
This document provides an overview of gene therapy. It discusses the basic concept of replacing a mutated gene with a healthy copy to cure diseases. It describes the central dogma of molecular biology and barriers to gene therapy like developing effective carriers or vectors. The main types of vectors discussed are viral vectors, including RNA virus vectors like retroviruses and DNA virus vectors like adenoviruses and adeno-associated viruses. Clinical trials of gene therapy are also mentioned, with the first using retroviruses to treat severe combined immunodeficiency. In conclusion, the document states that gene therapy requires identifying target genes, developing safe and efficient vectors, and conducting clinical trials optimized for specific diseases.
DNA lipofection - Efficiency in invitro and invivo transfectionpugazhenthi6
This document discusses DNA lipofection and its efficiency in in vitro and in vivo transfection. It begins by introducing gene therapy and its goal of introducing genes to prevent or cure diseases. It then describes the two main types of gene therapy: somatic cell gene therapy, which does not affect future generations; and germ line gene therapy, which is heritable but not currently attempted. The document outlines approaches to gene therapy, including ex vivo and in vivo methods. It discusses various vectors used in gene therapy such as viral vectors like retroviruses and adenoviruses and non-viral vectors like lipoplexes. The document concludes by noting both the disadvantages of potential immune responses and difficulties treating multiple gene disorders through gene therapy and
This document provides an overview of gene therapy. It defines gene therapy as using genes or oligonucleotide sequences as therapeutic molecules to treat genetic defects. The document describes the types of gene therapy, strategies used, methods of delivery including ex vivo and in vivo approaches, target cells, vectors, advantages and disadvantages. It also discusses the current status of gene therapy and diseases where successful clinical trials have been reported.
This document provides an overview of gene therapy, including what it is, different types and approaches, vectors used, methods of delivery, advantages and disadvantages. Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for a disease. It can be done via in vivo or ex vivo methods. Viral and non-viral vectors are used to deliver genes. While gene therapy holds promise to treat genetic diseases, it also faces challenges such as short-lived effects and safety issues.
This document provides an overview of gene therapy. It defines gene therapy as an experimental technique for correcting defective genes responsible for disease. It describes the main approaches like somatic cell gene therapy and germline gene therapy. It also discusses viral and non-viral vectors, delivery methods like in vivo and ex vivo, advantages like curing genetic diseases, and challenges like short-term effects and safety issues. Recent developments show promise for treating diseases like blindness and Parkinson's.
Gene therapy involves introducing genes into cells to treat disease. It works by correcting defective genes that cause diseases. The first approved gene therapy occurred in 1990 when a child with ADA-SCID was treated. There are two main types of gene therapy - somatic cell therapy targets body cells while germline therapy affects eggs and sperm. Significant challenges remain for gene therapy including developing safe methods for long-term effects and ensuring equitable access to new treatments. However, gene therapy has potential to cure many inherited diseases and revolutionize medicine.
Gene therapy is the process of inserting therapeutic genes into cells to prevent or cure wide range of diseases. The newly introduced genes will encode proteins and correct the deficiencies that occur in genetic diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease, and keep organism in good health. It is a technique for correcting defective genes responsible for disease and development.
Gene therapy involves inserting normal genes into a person's cells to treat a disease. There are several strategies for gene therapy, including ex vivo and in vivo methods. Viral vectors are commonly used to deliver therapeutic genes to target cells. However, gene therapy has faced challenges such as short-lived effects, immune responses, and safety issues with viral vectors. Continued research is working to overcome these issues and further develop promising applications of gene therapy.
Gene therapy advanced treatments for a new era aranca special reportAranca
Aranca's Report on Gene Therapy - a promising tool for Cancer, Parkinson's, HIV, severe combined immuno-deficiencies, hemophilia etc. In this report, you will discover the challenges associated with Gene Therapy as well as its expected future.
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
The document discusses gene therapy and its potential to treat genetic diseases. It describes how gene therapy works by introducing functional genes into cells to replace defective genes causing disease. The first approved gene therapy treated a girl with ADA-SCID by inserting a functional ADA gene. While promising, gene therapy faces challenges like short-lived effects and safety issues that must still be addressed.
Gene therapy involves modifying genes to treat or cure disease. It works by replacing mutated genes, inactivating abnormal genes, or introducing new genes. Early successes treated immune deficiencies, but challenges remain in achieving long-term effects without side effects. Promising areas are treating inherited retinal diseases and Parkinson's through localized delivery of therapeutic genes using viral or non-viral vectors. While offering potential cures, gene therapy also raises ethical issues that require ongoing discussion.
Emerging viral diseases pose a major threat and are becoming more common due to factors like globalization and urbanization. New technologies are helping address this issue, with genomic sequencing identifying viruses and rapid PCR diagnosis deployed in outbreak settings. Real-time PCR has been particularly useful for differentiating viral from bacterial infections during disease outbreaks. Continued development of antiviral drugs and vaccines remains an important focus, but rapid diagnostics can also help control disease spread through early case identification and contact tracing.
Similar to Overview and vectors for gene therapy (20)
Cyclic Peptides Current Status & Future Prospects.pdfDoriaFang
Cyclic peptides have potential advantages over linear peptides as drug candidates due to increased stability, binding affinity, and membrane permeability. Many cyclic peptides are approved drugs or in clinical trials. Recent trends include using modern technologies to discover novel cyclic peptides and developing peptide-drug conjugates to selectively deliver therapeutic payloads. Over 50 cyclic peptides have been approved, including pegcetacoplan which targets C3 complement proteins. Hundreds of cyclic peptides are in the research pipeline, and their prospects for drug development remain promising.
Antibody–Oligonucleotide Conjugates (AOCs) in Clinical Trials.pdfDoriaFang
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Greetings,
Hawk Energy is pleased to present you with the latest energy news
NewBase 20 June 2024 Energy News issue - 1731 by Khaled Al Awadi
Regards.
Founder & S.Editor - NewBase Energy
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MS & BS Mechanical Engineering (HON), USAGreetings,
Hawk Energy is pleased to present you with the latest energy news
NewBase 20 June 2024 Energy News issue - 1731 by Khaled Al Awadi
Regards.
Founder & S.Editor - NewBase Energy
Khaled M Al Awadi, Energy Consultant
MS & BS Mechanical Engineering (HON), USAGreetings,
Hawk Energy is pleased to present you with the latest energy news
NewBase 20 June 2024 Energy News issue - 1731 by Khaled Al Awadi
Regards.
Founder & S.Editor - NewBase Energy
Khaled M Al Awadi, Energy Consultant
MS & BS Mechanical Engineering (HON), USAGreetings,
Hawk Energy is pleased to present you with the latest energy news
NewBase 20 June 2024 Energy News issue - 1731 by Khaled Al Awadi
Regards.
Founder & S.Editor - NewBase Energy
Khaled M Al Awadi, Energy Consultant
MS & BS Mechanical Engineering (HON), USAGreetings,
Hawk Energy is pleased to present you with the latest energy news
NewBase 20 June 2024 Energy News issue - 1731 by Khaled Al Awadi
Regards.
Founder & S.Editor - NewBase Energy
Khaled M Al Awadi, Energy Consultant
MS & BS Mechanical Engineering (HON), USAGreetings,
Hawk Energy is pleased to present you with the latest energy news
NewBase 20 June 2024 Energy News issue - 1731 by Khaled Al Awadi
Regards.
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MS & BS Mechanical Engineering (HON), USA
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According to the World Economic Forum, digital technologies can help reduce global carbon emissions by up to 15%. However, digitalization also comes with some challenges. Thus, if we want to make a positive impact by increasing sustainability, we need to address challenges like the digital divide, energy consumption of IT, or the rise of electronic waste. In this talk, I want to explore how Agile can help to leverage Digital Sustainability.
Tired of chasing down expiring contracts and drowning in paperwork? Mastering contract management can significantly enhance your business efficiency and productivity. This guide unveils expert secrets to streamline your contract management process. Learn how to save time, minimize risk, and achieve effortless contract management.
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L'indice de performance des ports à conteneurs de l'année 2023SPATPortToamasina
Une évaluation comparable de la performance basée sur le temps d'escale des navires
L'objectif de l'ICPP est d'identifier les domaines d'amélioration qui peuvent en fin de compte bénéficier à toutes les parties concernées, des compagnies maritimes aux gouvernements nationaux en passant par les consommateurs. Il est conçu pour servir de point de référence aux principaux acteurs de l'économie mondiale, notamment les autorités et les opérateurs portuaires, les gouvernements nationaux, les organisations supranationales, les agences de développement, les divers intérêts maritimes et d'autres acteurs publics et privés du commerce, de la logistique et des services de la chaîne d'approvisionnement.
Le développement de l'ICPP repose sur le temps total passé par les porte-conteneurs dans les ports, de la manière expliquée dans les sections suivantes du rapport, et comme dans les itérations précédentes de l'ICPP. Cette quatrième itération utilise des données pour l'année civile complète 2023. Elle poursuit le changement introduit l'année dernière en n'incluant que les ports qui ont eu un minimum de 24 escales valides au cours de la période de 12 mois de l'étude. Le nombre de ports inclus dans l'ICPP 2023 est de 405.
Comme dans les éditions précédentes de l'ICPP, la production du classement fait appel à deux approches méthodologiques différentes : une approche administrative, ou technique, une méthodologie pragmatique reflétant les connaissances et le jugement des experts ; et une approche statistique, utilisant l'analyse factorielle (AF), ou plus précisément la factorisation matricielle. L'utilisation de ces deux approches vise à garantir que le classement des performances des ports à conteneurs reflète le plus fidèlement possible les performances réelles des ports, tout en étant statistiquement robuste.
L'indice de performance des ports à conteneurs de l'année 2023
Overview and vectors for gene therapy
1. Biopharma PEG https://www.biochempeg.com
Overview and Vectors For Gene Therapy
On January 25, 2022, Nature published an article listing seven technologies worthy of
attention this year. Targeted genetic therapies was on the list. The remaining six
technologies are: Fully finished genomes, Protein structure solutions, Quantum simulation,
Precise genome manipulation, Spatial multi-omics), CRISPR-based diagnostics.
An overview of gene therapy
Gene therapy refers to the delivery of exogenous genes with normal functions to target
cells in the human body with a certain carrier, and the purpose of treating diseases is
achieved by correcting the defective genes. If chemotherapy is a "symptom cure", then
gene therapy is a "cure for the root cause". With the gradual emergence of gene therapy
in clinical applications, it has become one of the most rapidly developing directions in the
field of biomedicine in recent years.
Most gene therapy is to obtain modified functional cells locally or in vitro, and then
transplant them into the patient. However, this method is limited in drug delivery and
cannot precisely deliver the drug to the target tissue. Of course, except for the liver,
because the liver has the function of filtering blood, in addition to intravenous infusion,
even subcutaneous injection can achieve specific targeted delivery to the liver. For gene
drug delivery in extrahepatic tissues, major pharmaceutical companies are making steady
progress to seize key technologies. Drug carrier is the key technology of gene therapy,
and commonly used carriers can be divided into viral vectors and non-viral vectors.
Gene Therapy Vectors
Depending on the source and nature of the vector, gene therapy vectors can be divided
into two categories: viral vectors and non-viral vectors. Viral vectors mainly include
2. Biopharma PEG https://www.biochempeg.com
lentivirus, adenovirus, retrovirus, adeno-associated virus, etc., and non-viral vectors
mainly include naked DNA, lipid carriers, polymer nanoparticles, and exosomes. Among
them, viral vectors are the main delivery vectors currently used, and about 70% of the
genetic drugs in clinical trials are viral vectors.
1. Viral Vectors
Viral vectors are the most commonly used vectors for gene therapy, mainly
because viral vectors can naturally infect cells. The genome of the virus includes a
coding region and a non-coding region. The genes in the coding region produce the
structural and non-structural proteins of the virus, while the non-coding region contains
cis-acting elements necessary for the replication and packaging of the virus. Gene
recombination technology can be used to modify the virus, eliminating the oncogene in the
genome and at the same time replicating the defective virus. Under normal circumstances,
in order to insert enough exogenous DNA into the virus, the unnecessary and essential
genes can also be deleted at the same time if necessary, so as to increase the capacity of
the viral vector for exogenous DNA.
Figure 1 Viral vectors for gene therapy
3. Biopharma PEG https://www.biochempeg.com
An ideal viral vector should have the following characteristics: it can encapsulate
exogenous genes and form virus particles; it can mediate the transfer and expression of
exogenous genes; it will not proliferate and spread in the environment, and will not cause
harm to the body. There are three main types of commonly used viral vectors:
Lentivirus (LV) vector, Adenovirus (ADV) vector and Adeno-associated virus (AAV)
vector. Adeno-associated virus is currently the most used vector.
(1) Lentiviral vectors
Lentivirus (LV) vectors are developed on the basis of HIV-1 (human immunodeficiency
virus type I). Lentiviral infection has the characteristics of integration, which can integrate
exogenous functional genes into the host chromosome, and the exogenous functional
genes become infectious virus particles under the action of virus encapsulation, so as to
achieve good gene therapy effect through stable and long-lasting expression.
After the lentiviral vector enters the cell, the carried genome is reverse transcribed into
DNA in the cytoplasm, and the reverse transcribed DNA enters the nucleus and integrates
into the cell genome. The DNA integrated into the cell genome can either generate small
RNA or be transcribed into mRNA for the expression of the target protein in the cytoplasm.
Lentivirus-mediated gene therapy can divide along with the division of the cell genome,
providing stable and efficient gene delivery.
(2) Adenovirus vectors
Adenovirus (ADV) vectors are the earliest human vector for gene delivery. Adenoviruses
are non-enveloped, double-stranded DNA, first isolated in the 1950s, and discovered in
the 1980s for gene delivery carrier potential. At present, no less than 60 types of
adenoviruses have been found, among which Ad5 (Adenovirus serotypes 5) type is
widely used as a gene delivery vector.
4. Biopharma PEG https://www.biochempeg.com
The delivery mechanism of adenovirus vectors is mainly receptor-mediated. The
recombinant adenovirus vectors enter the cell under receptor-mediated endocytosis, and
the genome carried by the adenovirus vectors enter the nucleus, but does not integrate
into the host cell genome, remaining outside the chromosome. Adenoviral vectors are
the most commonly used vaccine vectors and are less used in other areas.
(3) Adeno-associated virus vectors
Adeno-associated virus (AAV) vectors are non-integrated viral vectors (or the proportion
of gene integration ability is extremely low), which exist in an independent free form after
entering human cells and will not integrate into the host cell genome, thus reducing
related risks and showing good safety. Adeno-associated virus (ADV) is a class of
single-stranded DNA deficient viruses with the simplest structure. It has no envelope and
is shaped as naked 20-hedron particles. The scientific consensus is that it does not cause
any human disease and can infect different target organs according to the different serum.
Recombinant adeno-associated virus (rAAV) particles as gene therapy vectors have
successfully transduced mammalian cells since the early 1980s. Recombinant
adeno-associated virus particles bind to glycosylated receptors on the surface of host
cells, and enter cells to form endosome through clathrin-mediated endocytosis. The
subunit of viral capsid changes conformational changes after acidification, and the virus
carried by it disintegrates from the endosome and enters the nucleus. At this point, the
single-stranded DNA released from the capsid cannot be transcribed, requiring the
formation of double-stranded DNA with the assistance of DNA polymerase of the host
cell.
5. Biopharma PEG https://www.biochempeg.com
Figure 2 Approaches to gene therapy
Viral vectors have become a delivery tool for many gene therapies, and it has been
shown in many animal experiments that organ targeted gene delivery can be achieved
through high-throughput screening of suitable viral vectors and specific combination with
tissues. However, there are two difficult problems in viral vectors: one is that it is difficult
to mass produce viral vectors to meet the market demand; the other is that high drug dose
may stimulate immune response, leading to rapid degradation or neutralization of the
vector, and the safety needs to be further confirmed.
2. Non-viral vectors
The construction process of viral vectors is complicated and expensive, and after the viral
vectors enter the human body, immune responses will inevitably occur as the dose
increases, and some viruses even have off-target effects and carcinogenic risks. A series
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of unresolved problems limit the development of viral vectors. At the same time, non-viral
vector technology has developed rapidly in recent years as a vehicle for novel gene
delivery therapies. Compared with viral vectors, non-viral vectors have their unique
advantages: the use of natural or semi-synthetic compounds, lower toxicity and
immunogenicity, and biodegradable properties reduce the risk of gene therapy; non-viral
vectors can be engineered and engineered, improve the targeted delivery efficiency of the
vector; the non-viral vector is easy to produce and process transformation, and the cost is
controllable.
(1) Lipid nanoparticles
In the past more than a year, lipid nanoparticles have become one of the hottest drug
delivery vectors due to the approval of mRNA COVID-19 vaccines. Lipid nanoparticles
have previously been used as non-viral vectors for gene delivery by actively fusing with
lipid cell membranes for delivery into cells. Relevant studies have shown that lipid
nanoparticles can be used as a substitute for viral vectors and have great potential for
tissue-specific targeted delivery, which can be widely used in the delivery of RNA
vaccines, RNAi, antisense nucleic acids and other drug molecules.
At present, lipid nanoparticles are used for gene therapy. The lipid nanomaterials
encapsulate the genome and directly enter the target cells for in vivo treatment. They can
be efficiently delivered without relying on viral vectors, and at the same time reduce the
risk of viral vector insertion into carcinogenesis. With the development of nanotechnology,
it has been possible to systematically screen lipid nanoparticles, by changing their
composition, physicochemical properties and biological properties, to change the
distribution of the genome they carry in the organism. At the same time, lipid nanoparticles
are also combined with genetic engineering techniques to maximize the therapeutic effect.
(2) Polymer nanoparticles
7. Biopharma PEG https://www.biochempeg.com
Polymer nanoparticles, as gene delivery vehicles, are usually combined with gene
editing technology. For example, Sarepta Therapeutics' polymer nanoparticle delivery
platform (NanoGalaxy) combined with Sarepta's gene editing technology to develop a
novel gene editing therapy for the treatment of neuromuscular diseases. Preliminary in
vivo results show that polymer nanoparticles deliver genomes to specific muscle tissues
after systemic administration without the assistance of targeted delivery of viral vectors.
The polymer nanoparticle delivery platform contains thousands of polymers with different
chemical properties, which can be selected according to the different targets to be
reached, and carry different load genomes. The polymer nanoparticles of the NanoGalaxy
technology platform can deliver DNA, RNA or CRISPR gene editing systems.
Figure 3 Working principle of NanoGalaxy delivery technology platform
(3) Exosomes
Exosomes are discoid vesicles with a diameter of 40-160 nm wrapped in lipid bilayers.
They are derived from multivesicular bodies formed by the invagination of intracellular
lysosomal particles. The outer membrane of multivesicles is fused with the cell membrane.
After being released into the extracellular matrix, exosomes are natural carriers of
intercellular communication, which have been developed as drug delivery vehicles at
present.
8. Biopharma PEG https://www.biochempeg.com
Compared with other delivery systems, exosomes have the following
advantages: as multifunctional carriers, exosomes can encapsulate and deliver various
biological macromolecules such as small RNA, mRNA, DNA, and proteins; exosomes
have the ability to cross physiological barriers , and can even cross the blood-brain barrier;
exosomes can be genetically engineered to modify their surface proteins to achieve
targeted delivery to specific tissues, avoiding toxic side effects caused by accumulation in
non-essential organs.
At present, exosome-based targeted delivery gene therapy has shown the advantages of
enhanced efficacy and improved safety, and other companies have combined lipid
nanoparticles and exosomes to develop a new generation of non-viral gene therapy.
In the history of gene therapy delivery, various delivery methods have been developed.
From viral vectors to non-viral vectors, from adenovirus to exosomes, with the
advancement and development of science and technology, gene therapy also benefits. It
is believed that with the continuous innovation of technology, gene therapy will bring more
possibilities for drug development.
Biopharma PEG is a worldwide leader of PEG linker supplier. We have been focusing on
the development of a full range of medical applications and technologies for nanocarrier
systems, including various types of nanoparticles, liposomes, micelles, etc. We are
committed to providing a variety of PEG-liposome derivatives, including mPEG, DSPE
lipids with different molecular weight and functional PEG. We can provide the
following PEG products that used in COVID-19 mRNA vaccines.
1
mPEG-N,N-Ditetradecylacetamide
(ALC-0159)
CAS No.
1849616-42-7
2 mPEG-DMG CAS NO.
9. Biopharma PEG https://www.biochempeg.com
160743-62-4
3 mPEG-CH2CH2CH2-NH2 ---
4 mPEG-OH
CAS NO.:
9004-74-4
5 mPEG-CM (mPEG-AA) ---
6 mPEG-DSPE
CAS NO.:
147867-65-0
7 mPEG-DPPE
CAS NO.:
205494-72-0
References:
[1] Lentiviral Vector Pseudotypes: Precious Tools to Improve Gene Modification of Hematopoietic Cells
for Research and Gene Therapy.
[2] Adenovirus: the first effective in vivo gene delivery vector.
[3] AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.