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

Authors :: Solano L
Source :: JAAPA : official journal of the American Academy of Physician Assistants [JAAPA] 2024 Nov 01; Vol. 37 (11), pp. 17-22. Date of Electronic Publication: 2024 Oct 29.
Publication Year :: 2024
Abstract: 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.<br /> (Copyright © 2024 American Academy of Physician Associates.)

Subjects :: Humans
CRISPR-Cas Systems
Gene Editing methods
Anemia, Sickle Cell therapy
Anemia, Sickle Cell genetics
Genetic Therapy adverse effects
Genetic Therapy methods
Hemophilia A therapy
Hemophilia A genetics
Muscular Dystrophy, Duchenne therapy
Muscular Dystrophy, Duchenne genetics

Language :: English
ISSN :: 1547-1896
Volume :: 37
Issue :: 11
Database :: MEDLINE
Journal :: JAAPA : official journal of the American Academy of Physician Assistants
Publication Type :: Academic Journal
Accession number :: 39412239
Full Text :: https://doi.org/10.1097/01.JAA.0000000000000142

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