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Gene Therapy: Genes As Medicine

Gene therapy is the next generation of medicine targeting the underlying cause of a genetic disease. It has the potential to offer patients a transformational clinical benefit and dramatically improve quality of life.

GENE THERAPY: FREQUENTLY ASKED QUESTIONS

GENE THERAPY: BREAKTHROUGHS THAT CHANGE PATIENTS’ LIVES

Please click the image below to download the Gene Therapy Fact Sheet:

References:

1. NIH Genetics Home Reference. What is a Gene? https://ghr.nlm.nih.gov/primer/basics/gene. Accessed February 10, 2020.

2. Forbes. How Many Possible Combinations of DNA Are There? https://www.forbes.com/sites/quora/2017/01/20/how-many-possible-combinations-of-dna-are-there/. Accessed February 10, 2020.

3. NIH National Human Genome Research Institute. Genetic Disorders. https://www.genome.gov/For-Patients-and-Families/Genetic-Disorders. Accessed February 10, 2020.

4. NIH Genetics Home Reference. How Can Gene Mutations Affect Health and Development? https://ghr.nlm.nih.gov/primer/mutationsanddisorders/mutationscausedisease. Accessed February 10, 2020.

5. NIH Genetics Home Reference. How does Gene Therapy Work? https://ghr.nlm.nih.gov/primer/therapy/procedures. Accessed February 10, 2020.

6. NIH Genetics Home Reference. What is Gene Therapy? https://ghr.nlm.nih.gov/primer/therapy/genetherapy. Accessed February 10, 2020.

7. Data on file. Pfizer Inc, New York, NY.

8. Haasteren J, Hyde S, Gill D. Lessons Learned From Lung and Liver In-Vivo Gene Therapy: Implications for the Future. Expert Opin Biol Ther. 2018;18(9):959-972.

9. Gowing G, Svendsen S, Svendsen CS. Ex Vivo Gene Therapy for the Treatment of Neurological Disorders. Prog Brain Res. 2017;230:99-132.

10. NIH National Human Genome Research Institute. What is Genome Editing? https://www.genome.gov/about-genomics/policy-issues/what-is-Genome-Editing. Accessed February 10, 2020.

11. Carroll D. Genome Engineering with Zinc-Finger Nucleases. Genetics. 2011;188(4):773-782.

12. Unniyampurath U, Krishnan M, et al. RNA Interference in the Age of CRISPR: Will CRISPR Interfere with RNAi? Int J Mol Sci. 2016;17(3):291.

13. Your Genome. Facts: What is Gene Therapy? http://www.yourgenome.org/facts/what-isgene-therapy. Accessed February 10, 2020.

14. Arruda VR, Favaro P, Finn JD. Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy. Mol Ther. 2009;17(9):1492–1503.

15. Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood. 2013;122(1):23-36.

16. Payne J. Antibody and antigen tests. Patient Platform Limited Web site. https://patient.info/health/antibody-and-antigen-tests. Accessed February 10, 2020.