Gene therapy

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April 23, 2024
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  1. MedLine Plus. Genes and gene therapy. https://medlineplus.gov/genesandgenetherapy.html. Accessed Jan. 31, 2024.
  2. How gene therapy can cure or treat diseases. U.S. Food and Drug Administration. https://www.fda.gov/consumers/consumer-updates/how-gene-therapy-can-cure-or-treat-diseases. Accessed Feb. 8, 2024.
  3. Gene therapy basics. American Society of Gene + Cell Therapy. https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-basics. Accessed Jan. 31, 2024.
  4. What are genetic therapies? National Heart, Lung, and Blood Institute. https://www.nhlbi.nih.gov/health/genetic-therapies. Accessed Jan. 31, 2024.
  5. What is gene therapy? U.S. Food and Drug Administration. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy. Accessed Jan. 31, 2024.
  6. Flomenbergm P, et al. Overview of gene therapy, gene editing, and gene silencing. https://www.uptodate.com/contents/search. Accessed Jan. 31, 2024.
  7. Dara J, et al. Overview of gene therapy for inborn errors of immunity. https://www.uptodate.com/contents/search. Accessed Jan. 31, 2024.
  8. Kymriah (prescribing information). Novartis Pharmaceutical Corp.; 2022. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/kymriah-tisagenlecleucel. Accessed Jan. 31, 2024.
  9. Yescarta (prescribing information). Kite Pharma Inc.; 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/yescarta-axicabtagene-ciloleucel. Accessed Jan. 31, 2024.
  10. Luxturna (prescribing information). Spark Therapeutics Inc.; 2022. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna. Accessed Jan. 31, 2024.
  11. Zolgensma (prescribing information). Novartis Gene Therapies Inc.; 2019. https://www.fda.gov/vaccines-blood-biologics/zolgensma. Accessed Jan. 31, 2024.
  12. Imlygic (prescribing information). Amgen Inc.; 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/imlygic. Accessed Jan. 24, 2024.
  13. Shukla V, et al. The landscape of cellular and gene therapy products: Authorization, discontinuations and cost. Human Gene Therapy. Clinical Development. 2019; doi:10.1089/humc.2018.201.
  14. MedLine Plus. How does gene therapy work? https://medlineplus.gov/genesandgenetherapy.html. Accessed Jan. 31, 2024.
  15. Gene therapy — Frequently asked questions. National Organization for Rare Disorders Inc. https://rarediseases.org/gene-therapy/faqs. Accessed Jan. 31, 2024.
  16. Gene therapy — Frequently asked questions. Immune Deficiency Foundation. https://primaryimmune.org/understanding-primary-immunodeficiency/treatment/gene-therapy. Accessed Jan. 31, 2024.
  17. Deyle DR (expert opinion). Mayo Clinic. Feb. 8, 2024.
  18. Approved cellular and gene therapy products. U.S. Food and Drug Administration. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed Feb. 12, 2024.

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