MOLECULAR BASES OF THE GENOME
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This document discusses DNA, RNA, and the molecular bases of the genome. It covers how tracking DNA helps scientists trace the origins of genetic errors. RNA plays a role in genetic expression and manipulation of genetic material. Understanding the genome could allow it to function like a computer receiving orders from RNA. Studying the genome increases scientific understanding, but more remains to be learned about maintaining the genetic code and enzymes responsible for genetic processes. Understanding genetics could improve medical treatments and steer medicine towards prevention by addressing genetic predispositions.
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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
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(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.
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a proper description about the process microinjection and also about gene transfer. and different types of DNA delivery methods.
with advantages, disadvantages, limitations and applications.
Microarray technology
DNA microarrays allow researchers to analyze gene expression across an entire genome. Microarrays contain many copies of single genes arranged on a small chip. By detecting differences in gene expression levels between cell populations, microarrays can identify genes involved in processes like development, cancer, and cellular responses. Microarrays have been used to identify two subtypes of diffuse large B cell lymphoma with distinct gene expression profiles, which helps physicians predict patient outcomes and guide development of targeted cancer treatments.
GENE THERAPY
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
Gene therapy involves inserting functional genes into patients' cells to treat genetic diseases. It was first proposed in the 1960s and attempted in the 1970s. The first approved human gene therapy trial took place in 1990, treating a girl with severe combined immunodeficiency. Since then, gene therapy has been used experimentally to treat various diseases including cancer, cystic fibrosis, and HIV. There are two main approaches - ex vivo, where cells are removed and modified before being returned to the body, and in vivo, where genes are directly inserted into target tissues. Viruses are often used as vectors to deliver therapeutic genes, with retroviruses commonly used for ex vivo therapy and adenoviruses for in vivo.
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Gene therapy and gene delivery systems
Genes carry hereditary information and encode proteins. Gene therapy aims to treat diseases by correcting defective genes through various approaches like inserting a normal gene or repairing an abnormal gene. Early research in the 1980s involved inserting human genes into bacteria. The first human gene therapy treated a girl for SCID in 1990 by inserting a normal gene into her white blood cells. Viral vectors like retroviruses and adenoviruses as well as non-viral methods can deliver genes, but all methods face challenges like short-lived effects, immune responses, and difficulties treating multi-gene disorders and diseases. While progress has been made, setbacks include a death in 1999 from an immune response and some children developing cancer from viral vectors.
Researchers create the first model of the DNA ‘replication fork” and Sequen...
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Lesson 11 Gene Therapy
Gene therapy involves inserting a normal gene into a patient's cells to treat a genetic disorder, such as cystic fibrosis. Scientists are trying to develop gene therapy for cystic fibrosis by putting copies of the normal gene into patients' cells. Some early tests on cystic fibrosis patients showed improvements, but these did not last. In the future, gene therapy may be used to prevent genetic diseases by modifying genes in sex or egg cells so all cells have the correct version. However, gene therapy of sex cells is currently illegal.
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The seminar covers the experiments and inventions that led to the development of genome editing technologies. These inventions were derived from life itself: isolated from natural organisms and adapted for scientific and therapeutic goals. You will learn the history of how genome engineering tools, including CRISPR, and delivery technology, including AAV capsids, were created in their modern form. The seminar explores how genome editing and gene therapy technologies are giving individuals control over their own genomes, focusing on the treatment of genetic diseases. It will describe major companies and emerging trends in the gene therapy industry. Finally, the seminar will discuss how and where new discoveries, including accelerated algorithms for genetic engineering, will lead us in the near and distant future.
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Gene therapy: Where do we stand
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.
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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.
Gene therapy
This document discusses components of a gene therapy strategy for cancer treatment. It describes introducing exogenous DNA into cancer cells to alter gene expression and phenotype. There are two main aims - replacing defective tumor suppressor genes or introducing genes to kill cancer cells. Delivery methods include viral vectors, physical methods like injection, and chemical methods using liposomes. The document also discusses targeting tumor cells or normal cells like stem cells, using suicide genes or gene replacement, and overcoming challenges of systemic delivery.
Literary investigation into gene therapy in the treatment of cancer
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Microinjection
a proper description about the process microinjection and also about gene transfer. and different types of DNA delivery methods.
with advantages, disadvantages, limitations and applications.
Microarray technology
DNA microarrays allow researchers to analyze gene expression across an entire genome. Microarrays contain many copies of single genes arranged on a small chip. By detecting differences in gene expression levels between cell populations, microarrays can identify genes involved in processes like development, cancer, and cellular responses. Microarrays have been used to identify two subtypes of diffuse large B cell lymphoma with distinct gene expression profiles, which helps physicians predict patient outcomes and guide development of targeted cancer treatments.
GENE THERAPY
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
Gene therapy involves inserting functional genes into patients' cells to treat genetic diseases. It was first proposed in the 1960s and attempted in the 1970s. The first approved human gene therapy trial took place in 1990, treating a girl with severe combined immunodeficiency. Since then, gene therapy has been used experimentally to treat various diseases including cancer, cystic fibrosis, and HIV. There are two main approaches - ex vivo, where cells are removed and modified before being returned to the body, and in vivo, where genes are directly inserted into target tissues. Viruses are often used as vectors to deliver therapeutic genes, with retroviruses commonly used for ex vivo therapy and adenoviruses for in vivo.
Application of Whole Genome Sequencing in the infectious disease’ in vitro di...
This document discusses the application of whole genome sequencing in infectious disease diagnostics. It provides examples of how genome sequencing has been used to identify bacterial species, detect antibiotic resistance genes, and study outbreaks. The document also discusses challenges around regulatory approval of genomic tests, data sharing policies, and database management. Overall, it argues that whole genome sequencing is a valuable tool but that standards must be developed to ensure high quality data.
Gene therapy
This document provides an overview of gene therapy, including its types, approaches, vectors used, delivery methods, applications, advantages, and disadvantages. Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. The first approved gene therapy experiment treated a child for ADA-SCID. Somatic and germline gene therapy are the two main types. Vectors deliver therapeutic genes and include viral vectors like retroviruses and lentiviruses and non-viral methods. Applications show promise for diseases like cancer, cystic fibrosis, hemophilia, and neurological disorders. However, risks include toxicity, immune responses, and unpredictable effects that require further research.
Gene therapy and gene delivery systems
Genes carry hereditary information and encode proteins. Gene therapy aims to treat diseases by correcting defective genes through various approaches like inserting a normal gene or repairing an abnormal gene. Early research in the 1980s involved inserting human genes into bacteria. The first human gene therapy treated a girl for SCID in 1990 by inserting a normal gene into her white blood cells. Viral vectors like retroviruses and adenoviruses as well as non-viral methods can deliver genes, but all methods face challenges like short-lived effects, immune responses, and difficulties treating multi-gene disorders and diseases. While progress has been made, setbacks include a death in 1999 from an immune response and some children developing cancer from viral vectors.
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Developing tools & Methodologies for the NExt Generation of Genomics & Bio In...
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Gene delivery and gene therapy . Gene delivery , Gene therapy .
Gene delivery and gene therapy . Gene delivery , Gene therapy .
Literary investigation into gene therapy in the treatment of cancer
Literary investigation into gene therapy in the treatment of cancer
Application of Whole Genome Sequencing in the infectious disease’ in vitro di...
Application of Whole Genome Sequencing in the infectious disease’ in vitro di...
Similar to MOLECULAR BASES OF THE GENOME
Researchers create the first model of the DNA ‘replication fork” and Sequen...
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Dogma central
The DNA is the basis of our genetic code, we could almost say that we are all made of DNA; therefore all studies are trying to understand this important part of us. Over time, they have discovered that DNA contains all the instructions that control the development and function of every cell in our body. What we know is that the DNA is able to divide itself, replicating and giving two daughter strands which contain exactly the same information from DNA mother. Then these are transcribed into RNA and finally translated into proteins, this is what we know as the central dogma of genetic information.
Although nature seems to be so perfect there are some cases where this beautiful process fails, and this is where certain diseases are originated and can cause multiple problems. Scientists are increasingly closer to find answers and perhaps their studies can help in the future.
New treatments for Alzheimer's, autism and depression, could be developed.
It could be the starting point for future researches on genes involved in these diseases.
Knowing which genes are involved, people who are not sick yet, might prevent some disease.
These findings help us understand how diseases work and where they come from.
Encourages doctors and scientists to find more about this genes, to achieve excellent results that could benefit many people.
It gives us hope and determination to achieve incredible things in this medicine area; we know that humans are able to find and develop things that we have never imagine.
We know that DNA is the basis of everything, thus if we understand certain parts of it and what is involved on it, we would be able to control many diseases that affects society nowadays.
With these discovery we would be contributing to industry and researches.
new hypothesis could change the way we see things, and would make researchers focused in other cell structures such as ribosomes.
The cause of some diseases might not be in the DNA, but on the malfunctioning of ribosomes, in that way we must look for the real cause of them.
In my opinion this is a big step for medicine, although there is not yet a certain result, and they have to investigate more about the genes, they have a great part of the investigation that can guide them to find the solution to multiple diseases. I think that this kind of researches benefit a lot our society, because they are trying to improve people’s life, by finding the different places of de brain where illnesses are originated. With this project we can start thinking on possible cures and treatments for Alzheimer's, autism, depression and other disorders.
It's good to start investigating on other cellular structures that may be quite involved in the most complex processes of DNA. Scientists may have never wondered what real role of ribosome is. Thinking about new hypotheses and that maybe the ribosome is the central point is crazy but good, because they could be right.
Reparación material genético
Scientists have developed new methods to study DNA structure and mutations. One study showed how smoking can chemically alter DNA building blocks, leading to mutations, and researchers created artificial counterparts to detect specific mutation sites. Another found that DNA replication leaves about 1.5% of "scaffold" sequences unremoved, in regulatory gene switches, causing diseases. Understanding these scaffolds may help find disease-causing mutations, aiding treatment.
Genetics, the pillars for new discoveries
The document discusses two recent studies on genetics and genetic engineering. The first study developed a new type of restriction enzyme that recognizes longer DNA cutting sites, making it more specific. The second study identified a rare form of Congenital Adrenal Hyperplasia caused by an 11 beta hydroxilase deficiency linked to consanguineous marriages in some regions. Knowing the complete genetic profile of diseases can help develop improved treatments. A student comments that both studies will aid genetic research and modifying DNA to treat diseases.
The evolution of medicine: DNA
This document summarizes and comments on three scientific articles about recent discoveries related to DNA regulation and replication. The first article discusses a discovery about how metabolism is linked to epigenetic regulation of DNA through an enzyme that transfers acetyl groups. The second article discusses reconstituting the DNA replication fork to study its process and regulation. The third discusses the medical importance of understanding DNA replication and regulation to develop new disease treatments.
DNA structure
DNA is a long polymer of simple units called nucleotides. Each one contains a phosphate group (acid component), a sugar group (neutral component) and a nitrogen base (basic component).
Folding
Scientists have discovered new details of the biochemical interactions necessary for cell division. They captured images of the helicase enzyme that unwinds DNA as it gets drawn to and wraps around its target. Additionally, BCM scientists unraveled the mystery behind spontaneous DNA breaks in resting cells not undergoing replication. They determined that "R loops" consisting of hybrid RNA-DNA structures can produce double-strand breaks, explaining how DNA fragmentation can occur without external intervention. The findings provide a better understanding of essential biological processes like DNA replication and could enable new ways to fight cancer by controlling when cells divide.
Dna fingerprinting by shalini mishra..
Madhumita was pregnant at the time of her murder. Her fetus was sent for DNA fingerprinting to determine paternity and identify the perpetrator. DNA fingerprinting works by extracting DNA from a sample, amplifying it if needed, cutting it with restriction enzymes, separating the fragments by size using gel electrophoresis, blotting it onto a medium, treating it with a radioactive probe, and creating an image on x-ray film to view the individual's unique DNA pattern. DNA fingerprinting has applications in determining paternity, identifying criminals, studying genetic variability in endangered species, and detecting pathogens or GMOs.
FOLDING MOLECULAR BIOLOGY
LEIDY JOHANA HINCAPIÉ RESTREPO
MEDICINE STUDENT
Function of the recently discovered enhancer RNA molecules may open new avenues for gene therapy and Powerful technology of single cell RNA sequencing to track the genetic development of a human and a mouse Embryo.
1. presentation
2. Introduction, overview
3. Function of the recently discovered RNA molecules enhancer May open new avenues for gene therapy
4. Powerful technology of single cell RNA genetic sequencing to track the development of a human and a mouse Embryo
5. Utility for medicine
6. bibliography
folding molecular biology
This document summarizes two scientific studies on chromosome defects and genetic diseases. The first study discovered a chromosome therapy technique to correct severe chromosome defects by replacing an abnormal ring chromosome with a normal duplicated chromosome in stem cells. This could help repair birth defects. The second study found that analyzing chromatin architecture, the organization of DNA and proteins in chromosomes, could help identify regulatory DNA anomalies and better diagnose genetic diseases, since DNA sequencing alone is insufficient. This discovery paves the way for improved understanding and diagnosis of many genetic conditions.
The development of DNA sequencing Technology.pptx
This presentation discusses the development of DNA sequencing technology from early techniques to current and future innovations. It covers early methods like Maxam-Gilbert and Sanger sequencing that involved chemical modifications and gel electrophoresis. Next-generation sequencing allowed for massively parallel sequencing and fueled projects like the Human Genome Project. Third-generation techniques enabled faster, longer reads. Advancements have increased accuracy and speed while expanding applications in medicine, agriculture, and more. The future holds potential for sequencing that is faster, cheaper and more accessible to further genomic research.
Dna repair
This document summarizes two research articles about DNA repair and telomeres. The first article finds that telomeres get shorter with each cell division, protecting cells from premature senescence. It also finds that an RNA species called TERRA accumulates at critically short telomeres, signaling the cell to repair its DNA or stop dividing. The second article discovers that cancer cells may streamline their genomes by reducing redundant ribosomal DNA to proliferate more easily. Both articles highlight the importance of DNA repair mechanisms in regulating cell proliferation and preventing unwanted cell division like cancer.
Folding
DNA sequencing has been successfully used to diagnose previously difficult to identify mitochondrial diseases. It allowed for diagnosis of 10 children with mitochondrial diseases out of 100 tested, finding genetic mutations much faster than traditional methods. Additionally, a form of Z-DNA, not normally found at sites other than where DNA is being copied, has been discovered on nucleosomes through a process involving SWI/SNF proteins and ATP. This Z-nucleosome structure may provide new opportunities for protein binding and regulation of gene expression. DNA sequencing is an important medical advancement that can lead to faster diagnosis and earlier treatment of hard to identify diseases like mitochondrial disorders.
BTG 708 GENE THERAPY.pptx
A gene is an ordered sequence of nucleotides located in a particular position on a particular chromosome that encodes a specific functional product
New Insight Into the Human Genome Through the Lens of Evolution and decode M...
New Insight Into the Human Genome Through the Lens of Evolution and decode M...Christian Sanchez Valencia
DNA contains the genetic blueprint for human life. It can be used to diagnose diseases by comparing DNA sequences between past and present humans. DNA is transcribed into RNA, some of which encodes proteins and some that performs regulatory functions by determining gene expression. Scientists have discovered that up to 30% of human DNA is conserved at the RNA structure level through a process called transcription. Understanding how RNA is regulated through interactions with proteins and sequences is important for identifying patterns involved in diseases and their treatment.Similar to MOLECULAR BASES OF THE GENOME (20)
Researchers create the first model of the DNA ‘replication fork” and Sequen...
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Molecular switch that regulates DNA replication and tracking DNA
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Recently uploaded
Applied Science: Thermodynamics, Laws & Methodology.pdf
When I was asked to give a companion lecture in support of ‘The Philosophy of Science’ (https://shorturl.at/4pUXz) I decided not to walk through the detail of the many methodologies in order of use. Instead, I chose to employ a long standing, and ongoing, scientific development as an exemplar. And so, I chose the ever evolving story of Thermodynamics as a scientific investigation at its best.
Conducted over a period of >200 years, Thermodynamics R&D, and application, benefitted from the highest levels of professionalism, collaboration, and technical thoroughness. New layers of application, methodology, and practice were made possible by the progressive advance of technology. In turn, this has seen measurement and modelling accuracy continually improved at a micro and macro level.
Perhaps most importantly, Thermodynamics rapidly became a primary tool in the advance of applied science/engineering/technology, spanning micro-tech, to aerospace and cosmology. I can think of no better a story to illustrate the breadth of scientific methodologies and applications at their best.
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The Milky Way’s (MW) inner stellar halo contains an [Fe/H]-rich component with highly eccentric orbits, often referred to as the
‘last major merger.’ Hypotheses for the origin of this component include Gaia-Sausage/Enceladus (GSE), where the progenitor
collided with the MW proto-disc 8–11 Gyr ago, and the Virgo Radial Merger (VRM), where the progenitor collided with the
MW disc within the last 3 Gyr. These two scenarios make different predictions about observable structure in local phase space,
because the morphology of debris depends on how long it has had to phase mix. The recently identified phase-space folds in Gaia
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We also compare the observed phase-space distribution to FIRE-2 Latte simulations of GSE-like mergers, using a quantitative
measurement of phase mixing (2D causticality). The observed local phase-space distribution best matches the simulated data
1–2 Gyr after collision, and certainly not later than 3 Gyr. This is further evidence that the progenitor of the ‘last major merger’
did not collide with the MW proto-disc at early times, as is thought for the GSE, but instead collided with the MW disc within
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https://ashpc.eu/
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The cost of acquiring information by natural selection
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The cost of information acquisition by natural selection
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The binding of cosmological structures by massless topological defects
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equations sourced by a topological defect, i.e. a singularity of a very specific form, the result is a localized gravitational
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Compexometric titration/Chelatorphy titration/chelating titration
Classification
Metal ion ion indicators
Masking and demasking reagents
Estimation of Magnisium sulphate
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Masking agent, Demasking agents
formation of complex
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Instrumental Techniques-Photometry
Potentiometry
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Complex titration with EDTA.
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Applied Science: Thermodynamics, Laws & Methodology.pdf
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MOLECULAR BASES OF THE GENOME
- 1. MOLECULAR BASES OF THE GENOME Hernando Giraldo Pizarro Medicine Student
- 2. FOLDING
- 3. FOLDING
- 4. INTRODUCTION DNA and RNA molecules are of great importance to determine the basis of cell function , knowledge of their general open our way to understanding the different investigations
- 5. Tracking DNA helps scientist trace origins of genetics errors modification of simple molecules associated with DNA may help better handling of this Errors in our genes, our genetic material , are responsible for many hereditary diseases
- 6. Tracking DNA helps scientist trace origins of genetics errors • RNA is a viable answer to the question of simple molecule , with which you can manipulate the genetic material, only has a chain and is very similar to DNA
- 7. Tracking DNA helps scientist trace origins of genetics errors • RNA help in genetic expression, but so far not played a basic role in the activation of some specific gene, assertion that today change
- 8. Tracking DNA helps scientist trace origins of genetics errors • someday maybe the genome could function as a computer, receiving orders RNA, also providing better possibilities to genetic or diagnostic.
- 9. Tracking DNA helps scientist trace origins of genetics errors • Is very interesting to see how lately increases the effort of the scientific community by the knowledge of the genome, but not others, because that is the impulse of the human race, curiosity has always characterized us And what generates more intrigue know the "molecule" that defines "who I am”? No hurts to applaud this kind of work, because science routed towards a common good. .
- 10. Engineers devise genetic 'on' switch made exclusively of RNA DNA replication is a semi-conservative mechanism, through which an "identical" DNA strand is synthesized thus obtaining two chains is a step very susceptible to mistakes
- 11. Engineers devise genetic 'on' switch made exclusively of RNA • at some moments the mechanisms that drive fail and insert replication mistakes in DNA • The points more susceptible to this error are in which genes turn on and off
- 12. Engineers devise genetic 'on' switch made exclusively of RNA • is important to track polymerase enzymes which are responsible for dNTP unite to form the DNA strands generate a small but substantial amount of damage both.
- 13. Engineers devise genetic 'on' switch made exclusively of RNA • labelling enzymes responsible for the genetic processes becomes easier the detection of problems in the genome, in addition to making more precise knowledge of the genetic code
- 14. Engineers devise genetic 'on' switch made exclusively of RNA • show that many mutations occur in the main test sites that we still have much to learn from this genome, although a breakthrough still have a long way to go, recognizing enzymes responsible for maintaining the genetic code.
- 15. MEDICAL UTILITY
- 16. MEDICAL UTILITY • thanks to genetic studies more patients will have access to treatments for various diseases
- 17. MEDICAL UTILITY • problems as the response to external agents or genetic problems that could become concerns inherited from the past
- 18. MEDICAL UTILITY • meet our genetic predisposition may help steer more medicine in the way of prevention to stop pigeonholing medical staff diagnosed in medicine
- 19. BIBLIOGRAPHY • • MARTINEZ SÁNCHEZ, Lina María. Biología molecular. 7. ed. Medellín: UPB. Fac. de Medicina, 2012. 78 p. • •HARVEY,LODISH, biología celular y molecular 5.ed. Buenos Aires: Médica panamericana, 2006. 101p
- 20. Engineers devise genetic 'on' switch made exclusively of RNA • show that many mutations occur in the main test sites that we still have much to learn from this genome, although a breakthrough still have a long way to go, recognizing enzymes responsible for maintaining the genetic code.