Gene Augmentation of CHM Using Non-Viral Episomal Vectors in Models of Choroideremia

Choroideremia (CHM) is an X-linked chorioretinal dystrophy leading to progressive retinal degeneration that results in blindness by late adulthood. It is caused by mutations in the CHM gene encoding the Rab Escort Protein 1 (REP1), which plays a crucial role in the prenylation of Rab proteins ensuri...

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Detalles Bibliográficos
Autores principales: Toualbi, Lyes, Toms, Maria, Almeida, Patrick Vingadas, Harbottle, Richard, Moosajee, Mariya
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10607001/
https://www.ncbi.nlm.nih.gov/pubmed/37894906
http://dx.doi.org/10.3390/ijms242015225

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10607001/
https://www.ncbi.nlm.nih.gov/pubmed/37894906
http://dx.doi.org/10.3390/ijms242015225

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