Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies

Background and objectives: Choroideremia (CHM) is an X-linked recessive chorioretinal dystrophy caused by mutations involving the CHM gene. Gene therapy has entered late-phase clinical trials, although there have been variable results. This review gives a summary on the outcomes of phase I/II CHM ge...

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Detalles Bibliográficos
Autores principales: Abbouda, Alessandro, Avogaro, Filippo, Moosajee, Mariya, Vingolo, Enzo Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7826687/
https://www.ncbi.nlm.nih.gov/pubmed/33445564
http://dx.doi.org/10.3390/medicina57010064

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7826687/
https://www.ncbi.nlm.nih.gov/pubmed/33445564
http://dx.doi.org/10.3390/medicina57010064

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