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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

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