Person looking hopeful as text animates. Imagine scanning millions of lines of DNA and pinpointing a specific sequence of genetic code to open up possibilities. Possibilities propelled by precision.
Discover gene therapy

The potential of gene therapy could turn what if’s into what’s next for people living with serious diseases. Designed for healthcare professionals who treat patients with genetic conditions, this website provides educational information to help you better understand this rapidly advancing field.

Gain a foundation in gene therapy

Learn about the fundamentals of cell and genetic therapy, the different gene-therapy tools, and the range of delivery methods.

Discover gene therapy
DNA strand icon representing gene therapy methods
DNA strand icon representing gene therapy methods

Discover the field of gene editing

Review the latest technologies for gene editing—a type of gene therapy that aims to precisely target and modify a patient’s genes.10

Explore gene editing
ACGT letters graphic representing DNA code
ACGT letters graphic representing DNA code

Learn how gene addition works

Read about a type of gene therapy that seeks to add a functional copy of a faulty gene into the genome to affect protein expression.11-13

Understand gene addition
Two DNA strand icons joined by plus sign representing gene addition
Two DNA strand icons joined by plus sign representing gene addition

Get up to speed with more expert knowledge

This website is just one part of your learning journey. Dig deeper with links to published articles and other gene-therapy resources.

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Lab microscope icon representing gene therapy library
Lab microscope icon representing gene therapy library

Our commitment to you

Vertex Pharmaceuticals is committed to providing healthcare professionals with educational information about gene therapy.

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Stay with Vertex as we continue to explore the evolving field of gene therapy.

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References: 1. Jain S, Shukla S, Yang C, et al. TALEN outperforms Cas9 in editing heterochromatin target sites. Nat Commun. 2021;12(1):606. doi:10.1038/s41467-020-20672-5. 2. Maeder ML, Gersbach CA. Genome-editing technologies for gene and cell therapy. Mol Ther. 2016;24(3):430-446. doi:10.1038/mt.2016.10. 3. Simeon M, Dangwal S, Sachinidis A, Doss MX. Application of the pluripotent stem cells and genomics in cardiovascular research—what we have learnt and not learnt until now. Cells. 2021;10(11):3112. doi:10.3390/cells10113112. 4. Fathallah MD. Genomics, gene editing and biologics: the bolsters of the new medical paradigm. Curr Trends Biomedical Eng Biosci. 2017;6(4):1-4. doi:10.19080/CTBEB.2017.06.555694. 5. Sudhakar V, Richardson RM. Gene therapy for neurodegenerative diseases. Neurotherapeutics. 2019;16(1):166-175. doi:10.1007/s13311-018-00694-0. 6. Amado DA, Davidson BL. Gene therapy for ALS: a review. Mol Ther. 2021;29(12):3345-3358. doi:10.1016/j.ymthe.2021.04.008. 7. Dias MF, Joo K, Kemp JA, et al. Molecular genetics and emerging therapies for retinitis pigmentosa: basic research and clinical perspectives. Prog Retin Eye Res. 2018;63:107-131. doi:10.1016/j.preteyeres.2017.10.004. 8. Gene therapy. ClinicalTrials.gov. Accessed April 14, 2023. https://clinicaltrials.gov/ct2/results?cond=&term=gene+therapy&cntry=US&state=&city=&dist. 9. Approved cellular and gene therapy products. US Food & Drug Administration. Updated October 26, 2021. Accessed April 14, 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. 10. Li H, Yang Y, Hong W, Huang M, Wu M, Zhao X. Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects. Signal Transduct Target Ther. 2020;5(1):1. doi:10.1038/s41392-019-0089-y. 11. DeWeerdt S. Gene therapy: a treatment coming of age. Pharm J. 2014;293(7831). doi:10.1211/PJ.2014.20066677. 12. Dong AC, Rivella S. Gene addition strategies for ß-thalassemia and sickle cell anemia. Adv Exp Med Biol. 2017;1013:155-176. doi:10.1007/978-1-4939-7299-9_6. 13. Nowakowski A, Andrzejewska A, Janowski M, Walczak P, Lukomska B. Genetic engineering of stem cells for enhanced therapy. Acta Neurobiol Exp. 2013;73:1-18.