Novel gene therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A

Document Type

Article

Publication Title

Journal of the American Academy of Physician Assistants

Abstract

This article discusses novel genetic therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. Gene therapies have the potential to deliver more targeted and effective approaches to treatment, especially for rare diseases for which the availability of approved therapies is limited. This article describes the first FDA-approved crispr/ Cas9 treatment and the treatment protocols, indications, warnings, precautions, cost, and contraindications of four novel genetic therapies.

First Page

17

Last Page

22

DOI

10.1097/01.JAA.0000000000000142

Publication Date

11-1-2024

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