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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.
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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.
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Gene Therapy: Genes As Medicine | Pfizer
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