CRISPR in Cancer Biology and Therapy.pptx
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The document discusses the history and development of CRISPR-Cas9 as a genome editing tool. It describes how CRISPR was first discovered in bacteria in 1987 and how the Cas9 enzyme and guide RNAs were later characterized. It explains that CRISPR-Cas9 utilizes a naturally occurring bacterial immune system to induce targeted double-strand breaks in DNA directed by a programmable guide RNA. The document then discusses how CRISPR-Cas9 has been adapted from prokaryotes for use in eukaryotic genome editing and cancer research, as well as future directions for this technology.
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Application of crispr in cancer therapy
Many bacterial clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated (Cas) systems employ the dual RNA–guided DNA endonuclease Cas9 to defend against invading phages and conjugative plasmids by introducing site-specific double-stranded breaks in target DNA. Target recognition strictly requires the presence of a short protospacer adjacent motif (PAM) flanking the target site, and subsequent R-loop formation and strand scission are driven by complementary base pairing between the guide RNA and target DNA, Cas9–DNA interactions, and associated conformational changes. The use of CRISPR–Cas9 as an RNA-programmable
DNA targeting and editing platform is simplified by a synthetic single-guide RNA (sgRNA) mimicking the natural dual trans-activating CRISPR RNA (tracrRNA)–CRISPR RNA (crRNA) structure
Applications of Gene Editing: CRISPR-Cas9 in Cancer Therapeutics (Oncogenes)
This presentation is done by Group 8, for the module Gene and Tissue Culture Technology. In Taylors University Lakeside Campus, Selangor, Malaysia.
Crispar
This document provides an overview of the CRISPR-Cas immune system in bacteria and its applications. It discusses:
1. The history and components of the CRISPR-Cas system, including Cas proteins, CRISPR RNA, and protospacer adjacent motifs.
2. The three main types of CRISPR-Cas systems and their mechanisms of targeting DNA or RNA.
3. How engineered versions of Cas9 can be used for targeted genome editing and modulation of gene expression in mammalian cells.
4. Applications of CRISPR-Cas in microbiology such as genetic engineering of bacteria, gene repression/activation using deactivated Cas9, and developing sequence-specific antimicrobials.
Crispr cas
This document summarizes Jai Kishan's presentation on gene manipulation techniques. It discusses conventional plant breeding, mutation breeding, transgenic plants, and several precise gene editing techniques like ZFN, TALENs, and CRISPR-Cas9. CRISPR-Cas systems provide adaptive immunity in bacteria and archaea by recognizing foreign DNA. The document then reviews the history and components of the CRISPR-Cas system and its applications in cancer therapy, viral diseases, bacterial infections, and genetic disorders. It also discusses challenges like delivery methods, mosaicism, immune responses, and off-target mutations. Key references on the discovery of CRISPR and its use in agriculture and as a genetic manipulation tool are presented.
CRISPR/CAS9 ppt by sanjana pandey
This document describes the construction of a high capacity adenoviral vector (HCAdV) devoid of all viral genes that can be used to deliver the CRISPR/Cas9 system for genome editing. Researchers developed an intermediate shuttle plasmid containing the CRISPR/Cas9 genes and guide RNAs that could then be inserted into the HCAdV genome. The CRISPR/Cas9 system utilizes the natural bacterial immune system to precisely cut DNA at targeted locations guided by guide RNAs. This vector aims to improve the efficiency of delivering genome edited DNA to cells for applications such as gene therapy.
CRISPR Technology
A simple version of the CRISPR/Cas system, CRISPR/Cas9, has been modified to edit genomes. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed and/or new ones added.
CRISPR-Cas system
CRISPR (clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found within the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of bacteriophages that have previously infected the prokaryote and are used to detect and destroy DNA from similar phages during subsequent infections. Hence these sequences play a key role in the antiviral defense system of prokaryotes.
Cas9 (CRISPR-associated protein 9) is an enzyme that uses CRISPR sequences as a guide to recognize and cleave specific strands of DNA that are complementary to the CRISPR sequence. Cas9 enzymes together with CRISPR sequences form the basis of a technology known as CRISPR-Cas9 that can be used to edit genes within organisms.This editing process has a wide variety of applications including basic biological research, development of biotechnology products, and treatment of diseases.
The CRISPR-Cas system is a prokaryotic immune system that confers resistance to foreign genetic elements such as those present within plasmids and phages that provides a form of acquired immunity. RNA harboring the spacer sequence helps Cas (CRISPR-associated) proteins recognize and cut foreign pathogenic DNA. Other RNA-guided Cas proteins cut foreign RNA. CRISPR are found in approximately 50% of sequenced bacterial genomes and nearly 90% of sequenced archaea.
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Application of crispr in cancer therapy
Many bacterial clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated (Cas) systems employ the dual RNA–guided DNA endonuclease Cas9 to defend against invading phages and conjugative plasmids by introducing site-specific double-stranded breaks in target DNA. Target recognition strictly requires the presence of a short protospacer adjacent motif (PAM) flanking the target site, and subsequent R-loop formation and strand scission are driven by complementary base pairing between the guide RNA and target DNA, Cas9–DNA interactions, and associated conformational changes. The use of CRISPR–Cas9 as an RNA-programmable
DNA targeting and editing platform is simplified by a synthetic single-guide RNA (sgRNA) mimicking the natural dual trans-activating CRISPR RNA (tracrRNA)–CRISPR RNA (crRNA) structure
Applications of Gene Editing: CRISPR-Cas9 in Cancer Therapeutics (Oncogenes)
This presentation is done by Group 8, for the module Gene and Tissue Culture Technology. In Taylors University Lakeside Campus, Selangor, Malaysia.
Crispar
This document provides an overview of the CRISPR-Cas immune system in bacteria and its applications. It discusses:
1. The history and components of the CRISPR-Cas system, including Cas proteins, CRISPR RNA, and protospacer adjacent motifs.
2. The three main types of CRISPR-Cas systems and their mechanisms of targeting DNA or RNA.
3. How engineered versions of Cas9 can be used for targeted genome editing and modulation of gene expression in mammalian cells.
4. Applications of CRISPR-Cas in microbiology such as genetic engineering of bacteria, gene repression/activation using deactivated Cas9, and developing sequence-specific antimicrobials.
Crispr cas
This document summarizes Jai Kishan's presentation on gene manipulation techniques. It discusses conventional plant breeding, mutation breeding, transgenic plants, and several precise gene editing techniques like ZFN, TALENs, and CRISPR-Cas9. CRISPR-Cas systems provide adaptive immunity in bacteria and archaea by recognizing foreign DNA. The document then reviews the history and components of the CRISPR-Cas system and its applications in cancer therapy, viral diseases, bacterial infections, and genetic disorders. It also discusses challenges like delivery methods, mosaicism, immune responses, and off-target mutations. Key references on the discovery of CRISPR and its use in agriculture and as a genetic manipulation tool are presented.
CRISPR/CAS9 ppt by sanjana pandey
This document describes the construction of a high capacity adenoviral vector (HCAdV) devoid of all viral genes that can be used to deliver the CRISPR/Cas9 system for genome editing. Researchers developed an intermediate shuttle plasmid containing the CRISPR/Cas9 genes and guide RNAs that could then be inserted into the HCAdV genome. The CRISPR/Cas9 system utilizes the natural bacterial immune system to precisely cut DNA at targeted locations guided by guide RNAs. This vector aims to improve the efficiency of delivering genome edited DNA to cells for applications such as gene therapy.
CRISPR Technology
A simple version of the CRISPR/Cas system, CRISPR/Cas9, has been modified to edit genomes. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed and/or new ones added.
CRISPR-Cas system
CRISPR (clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found within the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of bacteriophages that have previously infected the prokaryote and are used to detect and destroy DNA from similar phages during subsequent infections. Hence these sequences play a key role in the antiviral defense system of prokaryotes.
Cas9 (CRISPR-associated protein 9) is an enzyme that uses CRISPR sequences as a guide to recognize and cleave specific strands of DNA that are complementary to the CRISPR sequence. Cas9 enzymes together with CRISPR sequences form the basis of a technology known as CRISPR-Cas9 that can be used to edit genes within organisms.This editing process has a wide variety of applications including basic biological research, development of biotechnology products, and treatment of diseases.
The CRISPR-Cas system is a prokaryotic immune system that confers resistance to foreign genetic elements such as those present within plasmids and phages that provides a form of acquired immunity. RNA harboring the spacer sequence helps Cas (CRISPR-associated) proteins recognize and cut foreign pathogenic DNA. Other RNA-guided Cas proteins cut foreign RNA. CRISPR are found in approximately 50% of sequenced bacterial genomes and nearly 90% of sequenced archaea.
SEMINAR ON CRISPR
1. CRISPR is a bacterial immune system that provides immunity against viruses. It works by matching DNA sequences from viruses to DNA spacers and cleaving invading DNA.
2. The type II CRISPR system is the most well studied and requires only three components - Cas9 protein, CRISPR RNA (crRNA), and trans-activating CRISPR RNA (tracrRNA) - to function. Combining crRNA and tracrRNA into a single RNA molecule called sgRNA was crucial for developing the CRISPR technique.
3. CRISPR technology has become a powerful genome editing tool due to its simplicity, high efficiency, low cost, and ease of use. It allows targeted
Gene therapy
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Crispr-Cas9 technology
Crispr-Cas9 system works on the concept of bacterial defence mechanism. The idea of which was replicated in eukaryotic cell in in- vitro condition by the researchers.
Crispr cas9
a brief description on the new emerging genome editing technology CRISPR-Cas9. this technique is making its place stronger and stronger day by day. and impossible things can be possible by this technique. and some main and famous names who discovered this technique.
Crispr cas9 based system for therapeutics
This document discusses the CRISPR-Cas9 system for genome editing and its applications. It provides a brief history of CRISPR's discovery and development as a tool. CRISPR-Cas9 uses CRISPR sequences and Cas9 nuclease to precisely target and edit DNA. The document outlines the basic components and mechanisms of CRISPR-Cas9, as well as its current applications in cancer immunotherapy, inhibiting angiogenesis, and correcting genetic disorders.
Review of CRISPR/Cas9
CRISPR/Cas9 is a powerful new technique for genome editing that allows DNA to be easily cut and modified. It involves using the Cas9 enzyme, guided by RNA, to create targeted double-stranded breaks in DNA which are then repaired, allowing the DNA sequence to be altered. This system was adapted from a bacterial immune system. CRISPR/Cas9 represents a major breakthrough as it is simpler, cheaper and more accurate than previous genome editing methods. It has already been used to edit genes in numerous organisms and holds promise for applications like correcting genetic diseases. However, off-target effects and ethical concerns surrounding its use in humans remain limitations that need to be addressed.
CRISPR - gene-editing for everyone
Have you considered that protein over-expression or inefficient mRNA knockdown may be masking physiological effects in your assays? Increasingly scientists are moving to endogenous gene-editing to characterise the function of their genes of interest.
Dr Chris Thorne from Cambridge Biotech Horizon Discovery discusses the ground breaking gene-editing technology CRISPR. The simplicity of experimental design has led to rapid adoption of the technology across the scientific community. However, challenges remain.
This Slidedeck focuses specifically on implementing CRISPR experiments, and explore a number of key considerations crucial to maximising chances of targeting success, whether your goal is to generate a knock-out or a knock-in. Chris also takes a look at some of the alternative uses of CRISPR, including sgRNA genome wide synthetic lethality screens.
The slides aim to support those researchers either planning to or already using CRISPR gene-editing in their lab. Horizon Discovery have also recently launched a program aimed specifically at academic cell biologists to promote the adoption of CRISPR by offering FREE CRISPR Reagents for knock-out cell line generation - more information available here. http://www.horizondiscovery.com/what-we-do/discovery-toolbox/genassist-crispr--raav-genome-editing-tools
Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemia
Gene therapy is the modern techniques of treatment of various diseases and disorders.
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases.
It is a technique for correcting defective genes responsible for disease development.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
The first approved gene therapy experiment occurred on September1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
Under the direction of William French Anderson, at the National Institutes of Health (NIH),
Crispr cas9 technology
This document provides an overview of CRISPR-Cas9 technology. It defines CRISPR as DNA sequences in bacteria that contain snippets of viral DNA used to detect and destroy invading viruses. The CRISPR system contains Cas9 proteins that cut DNA at specific locations guided by RNA, and RNA guide molecules. It works by integrating viral DNA into the bacteria's CRISPR loci, which are then transcribed into RNA to guide Cas9 to cleave invading viral DNA. Applications of CRISPR include disease modeling, cancer research, and correcting mutations in human embryos.
CRISPR/CAS9- THE GENE EDITING TOOL
The document provides an overview of the CRISPR/Cas9 gene editing technology. It discusses the history and components of the CRISPR system, how it works, applications in various fields like microbiology, biomedicine, agriculture, and therapeutics. Recent advances expand its use for transcriptional regulation, epigenetic editing, and live imaging. While powerful, it faces challenges like off-target effects that require further research to optimize its safe and ethical application.
TAPPING THE RNA WORLD FOR THERAPEUTICS
ASO drugs, Si-RNA, Delivery of si-RNA based drugs, CRISPR-Cas gene editing, mRNA based drugs, Aptamer based therapeutics, RNAI PATHWAY, Aptamer based delivery.
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CRISPR-Cas9 is a powerful gene editing tool that has promising applications in public health. It allows targeted editing of genes and could help treat diseases like HIV/AIDS, cancer, and antibiotic resistance. However, there are also ethical concerns about its use, such as off-target effects and questions around human enhancement. Going forward, CRISPR holds potential for developing new therapies and improving agriculture, but its applications will require addressing safety, consent, and access issues.
Crispr
An Introduction to Crispr Genome Editing
Crispr cas: A new tool of genome editing
CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats) are part of an adaptive defense mechanism in bacteria and archaea. Use of the CRISPR/Cas9 system for genome editing has been a major technological breakthrough, making genome modification in cells or organisms fast, more efficient, and much more robust than previous genome editing methods. Single guide RNAs (sgRNAs) or guide RNAs (gRNAs) direct and activate the Cas9 endonuclease at a specific genomic sequence. Cas9 then cleaves the target DNA, making it available for repair by the non-homologous end joining (NHEJ) system or for creating an insertion site for exogenous donor DNA by homologous recombination.
Crispr technique
Genome editing uses engineered nucleases to insert, delete, or replace sections of the genome. CRISPR/Cas9 is a popular genome editing technique that uses guide RNA to direct nucleases to specific DNA sequences. While promising for treating disease, human germline editing raises ethical concerns. Early studies editing human embryos and non-viable embryos demonstrated proof-of-concept but had low efficiencies and off-target mutations. Later studies improved targeting and showed potential for correcting genetic defects. However, regulation is needed as the first claimed use of CRISPR to genetically edit human babies was unapproved.
Gene delivery and gene therapy . Gene delivery , Gene therapy .
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.
Crispr cas9 ( a overview)
this slide provides an insight on newly emerging genome editing technique CRISPR Cas9 mechanism and applications.
Crispr cas
CRISPR-Cas is a natural defense system in bacteria that uses CRISPR sequences and Cas proteins to target and degrade foreign DNA such as from viruses. It has been adapted for genome editing in other organisms using a Cas9 protein guided by a synthetic single guide RNA to introduce targeted double-strand breaks. This system allows for precise genome modifications and has applications in biomedical research, disease treatment, and engineering of plants and other organisms. However, off-target effects and delivery methods require further optimization.
Crispr cas9
This document discusses the CRISPR-Cas9 genome editing technique. It begins with an overview of genome editing and provides a brief history. It then focuses on explaining CRISPR-Cas9, including its key components, how it was discovered as a natural bacterial immune system, and how it functions as a genomic tool. The document outlines the general CRISPR-Cas9 protocol and recent advances in the technique. It discusses applications in agriculture and for diseases. It also touches on advantages and limitations, as well as ethical issues. Two case studies are provided that demonstrate using CRISPR-Cas9 to modify genes in rice plants.
CRISPR-Cas systems and applications
i explained about basics of genome engineering and crispr system.
CRISPR will change the world and it is just the beginning, are you ready to meet the future? you think its great and beautiful or.....?
please give your feedback to my email
pooyanaghshbandi@yahoo.com
i am starting to write a critical and fantastic review article about CRISPR, if you are interested to join please contact me.
Genome Editing Review - Recent Technologies & Potential Applications
In this SlideShare, we'll recap the basics of gene editing and cover important basics such as the terminology, forward vs reverse genetics, as well as mechanisms of gene editing (homology-directed & non-homologous end joining). We'll also compare the popular gene-editing platforms including ZFNs, TALENs and CRISPR/Cas system, along with the potential applications of gene editing from clinical therapy to food sustainability.
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This document provides an overview of the CRISPR-Cas9 genome editing system. It discusses the history and mechanism of genome editing, describes the key components and mechanism of CRISPR-Cas9, and outlines its applications in research including in animal models like zebrafish. The document also discusses advantages and disadvantages of CRISPR-Cas9 as well as ethical issues and future prospects of this genome editing technique.Crispr technology
Researchers from Columbia University converted a bacterial immune system into the world's smallest data recorder. They modified E. coli bacteria to take advantage of the CRISPR-Cas system, which allows bacteria to record interactions with their environment. The scientists hijacked the CRISPR gene function and modified it to record external signals. This could allow bacteria swallowed in patients to record biological changes throughout the digestive tract, enabling disease diagnosis and environmental monitoring at the microscopic level without disruption.
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SEMINAR ON CRISPR
1. CRISPR is a bacterial immune system that provides immunity against viruses. It works by matching DNA sequences from viruses to DNA spacers and cleaving invading DNA.
2. The type II CRISPR system is the most well studied and requires only three components - Cas9 protein, CRISPR RNA (crRNA), and trans-activating CRISPR RNA (tracrRNA) - to function. Combining crRNA and tracrRNA into a single RNA molecule called sgRNA was crucial for developing the CRISPR technique.
3. CRISPR technology has become a powerful genome editing tool due to its simplicity, high efficiency, low cost, and ease of use. It allows targeted
Gene therapy
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Crispr-Cas9 technology
Crispr-Cas9 system works on the concept of bacterial defence mechanism. The idea of which was replicated in eukaryotic cell in in- vitro condition by the researchers.
Crispr cas9
a brief description on the new emerging genome editing technology CRISPR-Cas9. this technique is making its place stronger and stronger day by day. and impossible things can be possible by this technique. and some main and famous names who discovered this technique.
Crispr cas9 based system for therapeutics
This document discusses the CRISPR-Cas9 system for genome editing and its applications. It provides a brief history of CRISPR's discovery and development as a tool. CRISPR-Cas9 uses CRISPR sequences and Cas9 nuclease to precisely target and edit DNA. The document outlines the basic components and mechanisms of CRISPR-Cas9, as well as its current applications in cancer immunotherapy, inhibiting angiogenesis, and correcting genetic disorders.
Review of CRISPR/Cas9
CRISPR/Cas9 is a powerful new technique for genome editing that allows DNA to be easily cut and modified. It involves using the Cas9 enzyme, guided by RNA, to create targeted double-stranded breaks in DNA which are then repaired, allowing the DNA sequence to be altered. This system was adapted from a bacterial immune system. CRISPR/Cas9 represents a major breakthrough as it is simpler, cheaper and more accurate than previous genome editing methods. It has already been used to edit genes in numerous organisms and holds promise for applications like correcting genetic diseases. However, off-target effects and ethical concerns surrounding its use in humans remain limitations that need to be addressed.
CRISPR - gene-editing for everyone
Have you considered that protein over-expression or inefficient mRNA knockdown may be masking physiological effects in your assays? Increasingly scientists are moving to endogenous gene-editing to characterise the function of their genes of interest.
Dr Chris Thorne from Cambridge Biotech Horizon Discovery discusses the ground breaking gene-editing technology CRISPR. The simplicity of experimental design has led to rapid adoption of the technology across the scientific community. However, challenges remain.
This Slidedeck focuses specifically on implementing CRISPR experiments, and explore a number of key considerations crucial to maximising chances of targeting success, whether your goal is to generate a knock-out or a knock-in. Chris also takes a look at some of the alternative uses of CRISPR, including sgRNA genome wide synthetic lethality screens.
The slides aim to support those researchers either planning to or already using CRISPR gene-editing in their lab. Horizon Discovery have also recently launched a program aimed specifically at academic cell biologists to promote the adoption of CRISPR by offering FREE CRISPR Reagents for knock-out cell line generation - more information available here. http://www.horizondiscovery.com/what-we-do/discovery-toolbox/genassist-crispr--raav-genome-editing-tools
Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemia
Gene therapy is the modern techniques of treatment of various diseases and disorders.
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases.
It is a technique for correcting defective genes responsible for disease development.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
The first approved gene therapy experiment occurred on September1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
Under the direction of William French Anderson, at the National Institutes of Health (NIH),
Crispr cas9 technology
This document provides an overview of CRISPR-Cas9 technology. It defines CRISPR as DNA sequences in bacteria that contain snippets of viral DNA used to detect and destroy invading viruses. The CRISPR system contains Cas9 proteins that cut DNA at specific locations guided by RNA, and RNA guide molecules. It works by integrating viral DNA into the bacteria's CRISPR loci, which are then transcribed into RNA to guide Cas9 to cleave invading viral DNA. Applications of CRISPR include disease modeling, cancer research, and correcting mutations in human embryos.
CRISPR/CAS9- THE GENE EDITING TOOL
The document provides an overview of the CRISPR/Cas9 gene editing technology. It discusses the history and components of the CRISPR system, how it works, applications in various fields like microbiology, biomedicine, agriculture, and therapeutics. Recent advances expand its use for transcriptional regulation, epigenetic editing, and live imaging. While powerful, it faces challenges like off-target effects that require further research to optimize its safe and ethical application.
TAPPING THE RNA WORLD FOR THERAPEUTICS
ASO drugs, Si-RNA, Delivery of si-RNA based drugs, CRISPR-Cas gene editing, mRNA based drugs, Aptamer based therapeutics, RNAI PATHWAY, Aptamer based delivery.
Crispr future prospects in public health
CRISPR-Cas9 is a powerful gene editing tool that has promising applications in public health. It allows targeted editing of genes and could help treat diseases like HIV/AIDS, cancer, and antibiotic resistance. However, there are also ethical concerns about its use, such as off-target effects and questions around human enhancement. Going forward, CRISPR holds potential for developing new therapies and improving agriculture, but its applications will require addressing safety, consent, and access issues.
Crispr
An Introduction to Crispr Genome Editing
Crispr cas: A new tool of genome editing
CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats) are part of an adaptive defense mechanism in bacteria and archaea. Use of the CRISPR/Cas9 system for genome editing has been a major technological breakthrough, making genome modification in cells or organisms fast, more efficient, and much more robust than previous genome editing methods. Single guide RNAs (sgRNAs) or guide RNAs (gRNAs) direct and activate the Cas9 endonuclease at a specific genomic sequence. Cas9 then cleaves the target DNA, making it available for repair by the non-homologous end joining (NHEJ) system or for creating an insertion site for exogenous donor DNA by homologous recombination.
Crispr technique
Genome editing uses engineered nucleases to insert, delete, or replace sections of the genome. CRISPR/Cas9 is a popular genome editing technique that uses guide RNA to direct nucleases to specific DNA sequences. While promising for treating disease, human germline editing raises ethical concerns. Early studies editing human embryos and non-viable embryos demonstrated proof-of-concept but had low efficiencies and off-target mutations. Later studies improved targeting and showed potential for correcting genetic defects. However, regulation is needed as the first claimed use of CRISPR to genetically edit human babies was unapproved.
Gene delivery and gene therapy . Gene delivery , Gene therapy .
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.
Crispr cas9 ( a overview)
this slide provides an insight on newly emerging genome editing technique CRISPR Cas9 mechanism and applications.
Crispr cas
CRISPR-Cas is a natural defense system in bacteria that uses CRISPR sequences and Cas proteins to target and degrade foreign DNA such as from viruses. It has been adapted for genome editing in other organisms using a Cas9 protein guided by a synthetic single guide RNA to introduce targeted double-strand breaks. This system allows for precise genome modifications and has applications in biomedical research, disease treatment, and engineering of plants and other organisms. However, off-target effects and delivery methods require further optimization.
Crispr cas9
This document discusses the CRISPR-Cas9 genome editing technique. It begins with an overview of genome editing and provides a brief history. It then focuses on explaining CRISPR-Cas9, including its key components, how it was discovered as a natural bacterial immune system, and how it functions as a genomic tool. The document outlines the general CRISPR-Cas9 protocol and recent advances in the technique. It discusses applications in agriculture and for diseases. It also touches on advantages and limitations, as well as ethical issues. Two case studies are provided that demonstrate using CRISPR-Cas9 to modify genes in rice plants.
CRISPR-Cas systems and applications
i explained about basics of genome engineering and crispr system.
CRISPR will change the world and it is just the beginning, are you ready to meet the future? you think its great and beautiful or.....?
please give your feedback to my email
pooyanaghshbandi@yahoo.com
i am starting to write a critical and fantastic review article about CRISPR, if you are interested to join please contact me.
Genome Editing Review - Recent Technologies & Potential Applications
In this SlideShare, we'll recap the basics of gene editing and cover important basics such as the terminology, forward vs reverse genetics, as well as mechanisms of gene editing (homology-directed & non-homologous end joining). We'll also compare the popular gene-editing platforms including ZFNs, TALENs and CRISPR/Cas system, along with the potential applications of gene editing from clinical therapy to food sustainability.
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Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemia
Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemia
Gene delivery and gene therapy . Gene delivery , Gene therapy .
Gene delivery and gene therapy . Gene delivery , Gene therapy .
Genome Editing Review - Recent Technologies & Potential Applications
Genome Editing Review - Recent Technologies & Potential Applications
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This document provides information about CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) including:
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3) There is an ongoing patent dispute between researchers Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang over who first demonstrated using CRISPR-Cas9 for genome editing in human cells.
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CRISPR-Cas9 is a gene editing technique that utilizes the Cas9 enzyme to cut DNA at specific locations guided by CRISPR RNA. It allows scientists to precisely modify genes and has applications in medicine, agriculture, and scientific research. Some examples include developing disease-resistant crops and mosquitoes, growing human organs in pigs, and potentially curing genetic diseases. While promising, CRISPR also faces ethical concerns regarding safety, unintended effects, germline editing, and unequal access to treatment. Overall, CRISPR is a revolutionary new biotechnology but more research is still needed to fully realize its benefits and address ethical implications.
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- 1. CRISPR in Cancer Biology and Therapy Omoyayi Ibrahim 20174831@std.neu.edu.tr Department of Biomedical Engineering Near East University
- 2. History of CRISPR CRISPRs were first identified in E. coli in 1987 by a Japanese scientist, Yoshizumi Ishino, and his team, who accidentally cloned an unusual series of repeated sequences interspersed with spacer sequences while analyzing a gene responsible for the conversion of alkaline phosphatase. Emmanuelle Charpentier is the co-inventor of CRISPR. Together with Dr. Doudna, Dr. Charpentier was involved in the biochemical characterization of guide RNA and Cas9 enzyme- mediated DNA cleavage
- 3. Introduction to CRISPR Cas 9 System CRISPR stands for Clustered Regularly Interspaced Palindromic Repeats. CRISPR-Cas9 was adapted from a naturally occurring genome editing system that bacteria use as an immune defense. DNA endonuclease that is directed to induce double strand breaks (DSBs) at specific genomic loci via a programmable guide RNA (gRNA) molecule that mediates complementary DNA–RNA base pairing. The target sequence must be flanked on the 3′ side by an ‘NGG’ protospacer adjacent motif (PAM) sequence. The DSB created by Cas9 can be resolved by either precise homology-directed repair (HDR) or, non- homologous end joining (NHEJ) HDR enables the introduction of specific changes, while insertions and deletions (indels) from NHEJ can be exploited to disrupt coding and noncoding sequences
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- 5. Adaptation of CRISPR / CAS 9 from Prokaryotes into a Therapy in Eukaryotes Nature Review, 2020
- 6. The development of CRISPR tools that can be applied to the study of cancer biology Nature Review, 2020
- 7. Future Direction on CRISPR Cas 9 System Nature Review, 2020
- 8. References 1. Sung, H. et al. Global Cancer Statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J. Clin. 71, 209–249 (2021). 2. Mojica, F. J., Díez-Villaseñor, C., García-Martínez, J. & Soria, E. Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements. J. Mol. Evol. 60, 174–182 (2005). 3. Barrangou, R. et al. CRISPR provides acquired resistance against viruses in prokaryotes. Science 315, 1709–1712 (2007). 4. Bolotin, A., Quinquis, B., Sorokin, A. & Ehrlich, S. D. Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin. Microbiology 151, 2551–2561 (2005). 5. Pourcel, C., Salvignol, G. & Vergnaud, G. CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies. Microbiology 151, 653–663 (2005). 6. Cho, S. W., Kim, S., Kim, J. M. & Kim, J.-S. Targeted genome engineering in human cells with the Cas9 RNA- guided endonuclease. Nat. Biotechnol. 31, 230–232 (2013). 7. Jinek, M. et al. RNA-programmed genome editing in human cells. eLife 2, e00471 (2013). 8. Mali, P. et al. RNA-guided human genome engineering via Cas9. Science 339, 823–826 (2013). 9. Cong, L. et al. Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819–823 (2013). 10. Adli, M. The CRISPR tool kit for genome editing and beyond. Nat. Commun. 9, 1911 (2018)
- 9. Questions? Feel free to email me any questions you have; 20174831@std.neu.edu.tr