Novartis Gene Therapies Summer Intern Program 12 weeks 9 ...
Gene Therapy - Novartis
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What are genes? Gene Therapy Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body 1 . Every person has two copies of most of their genes - one from each parent 1 . Cell Nucleus Chromosome DNA Gene Every person has around 20 000 genes small variations in genes result in differences in people’s appearance and, potentially, health 1 . What are genetic diseases? Genetic diseases happen when a critical piece or whole section of DNA is substituted, deleted or duplicated 2 . These changes are called genetic mutations 3 . Well-known genetic diseases 4 : Substitution Original DNA Sequence Genetic mutations Deletion Duplication Cystic fibrosis Hemophilia Sickle cell anemia While some mutations are harmless, others can cause serious genetic diseases that are passed to future generations 4 . What is gene therapy? Gene therapy aims to treat or prevent a wide array of genetic diseases by suppressing, replacing or augmenting mutated genes with functioning copies 5 . New genes are transferred into cells using transporters known as vectors, which are often made from modified, inactivated viruses 5 . Gene therapy and cell therapy are overlapping fields of biomedical research and treatment, but they are not the same 7 . The gene is inserted or altered directly in the patient New gene is packaged inside vector New gene is packaged inside vector There are two ways to deliver gene therapy 5 The gene is modified or replaced in cells that have been removed from the body and that are then returned to the patient ex vivo in vivo What is the difference between gene therapy and cell therapy? Cell therapy aims to treat diseases by restoring or augmenting certain sets of cells or by using cells to carry a therapy through the body 7 . Gene therapies aim to treat or prevent diseases by modifying, repairing or replacing a mutated gene 7 . Why is gene therapy important? It is estimated that millions of people globally suffer from more than 10 000 rare diseases caused by mutation of a single gene 4 . For these patients, gene therapy offers hope, through its potential to treat previously untreatable diseases across a wide array of medical fields 8 : Neurology Ophthalmology Cardiovascular diseases References 1. NIH U.S. National library of medicine. What is a gene? Available at: https://ghr.nlm.nih. gov/primer/basics/gene. Last accessed July 2018. 2. NIH U.S. National library of medicine. What kinds of gene mutations are possible? Available at: https://ghr.nlm.nih.gov/primer/mutationsanddisorders/possiblemutations. Last accessed July 2018. 3. NIH. U.S. National library of medicine. What is a gene mutation and how do mutations occur? Available at: https://ghr.nlm.nih.gov/primer/mutationsanddisorders/ genemutation. Last accessed July 2018. 4. WHO. Genes and human disease. Available at: http://www.who.int/genomics/public/ geneticdiseases/en/index2.html. Last accessed July 2018. 5. High KA. The Jeremiah Metzger Lecture: Gene Therapy for Inherited Disorders: From Christmas Disease to Leber’s Amaurosis. Transactions of the American Clinical and Climatological Association. 2009; 120: 331-359. 6. Friedman T. A brief history of gene therapy. Nature Genetics. 1992; 2: 93-98. 7. American Society of Gene & Cell Therapy. Gene and cell therapy designed. Available at: www.asgct.org/about_gene_therapy/genevscell.php. Last accessed June 2018. 8. O’Reilly M, Kohn DB, Bartlett J, et al. Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012. Human Gene Therapy. 2013; 24(4): 355-362. Scientists have been researching gene therapy for over 50 years 6 . New gene packaged inside vector Vector New gene
Transcript of Gene Therapy - Novartis
What are genes?
Gene Therapy
Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body1. Every person has two copies of most of their genes - one from each parent1.
Cell
Nucleus
Chromosome DNA
Gene
Every person has around
20 000 genes small variations in genes result in differences in people’s appearance and, potentially, health1.
What are genetic diseases?Genetic diseases happen when a critical piece or whole section of DNA is substituted, deleted or duplicated2. These changes are called genetic mutations3.
Well-known genetic diseases4:
SubstitutionOriginal
DNA SequenceGenetic mutations
Deletion Duplication
Cystic fibrosis Hemophilia Sickle cell anemia
While some mutations are harmless, others can cause serious genetic diseases that are passed to future generations4.
What is gene therapy?Gene therapy aims to treat or prevent a wide array of genetic diseases by suppressing, replacing or augmenting mutated genes with functioning copies5. New genes are transferred into cells using transporters known as vectors, which are often made from modified, inactivated viruses5.
Gene therapy and cell therapy are overlapping fields of biomedical research and treatment, but they are not the same 7.
The gene is inserted or altered directly in the patient
New gene is packaged inside vector
New gene is packaged inside vector
There are two ways to deliver gene therapy5
The gene is modified or replaced in cells that have been removed from the body and that are then returned to the patient
ex vivo in vivo
What is the difference between gene therapy and cell therapy?
Cell therapy aims to treat diseases by restoring or augmenting certain sets of cells or by using cells to carry a therapy through the body7.
Gene therapies aim to treat or prevent diseases by modifying, repairing or replacing a mutated gene7.
Why is gene therapy important?It is estimated that millions of people globally suffer from more than 10 000 rare diseases caused by mutation of a single gene4.
For these patients, gene therapy offers hope, through its potential to treat previously untreatable diseases across a wide array of medical fields8:
NeurologyOphthalmology Cardiovascular diseases
References1. NIH U.S. National library of medicine. What is a gene? Available at: https://ghr.nlm.nih.
gov/primer/basics/gene. Last accessed July 2018.
2. NIH U.S. National library of medicine. What kinds of gene mutations are possible? Available at: https://ghr.nlm.nih.gov/primer/mutationsanddisorders/possiblemutations. Last accessed July 2018.
3. NIH. U.S. National library of medicine. What is a gene mutation and how do mutations occur? Available at: https://ghr.nlm.nih.gov/primer/mutationsanddisorders/genemutation. Last accessed July 2018.
4. WHO. Genes and human disease. Available at: http://www.who.int/genomics/public/geneticdiseases/en/index2.html. Last accessed July 2018.
5. High KA. The Jeremiah Metzger Lecture: Gene Therapy for Inherited Disorders: From Christmas Disease to Leber’s Amaurosis. Transactions of the American Clinical and Climatological Association. 2009; 120: 331-359.
6. Friedman T. A brief history of gene therapy. Nature Genetics. 1992; 2: 93-98.
7. American Society of Gene & Cell Therapy. Gene and cell therapy designed. Available at: www.asgct.org/about_gene_therapy/genevscell.php. Last accessed June 2018.
8. O’Reilly M, Kohn DB, Bartlett J, et al. Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012. Human Gene Therapy. 2013; 24(4): 355-362.
Scientists have been researching
gene therapy for over
50 years6.New gene packaged inside vector
Vector
New gene
