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Article

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

International journal of molecular sciences - 16 Oct 2023

Toualbi Lyes, Toms Maria, Almeida Patrick Vingadas, Harbottle Richard, Moosajee Mariya

Abstract excerpt

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 ensuring correct intracellular trafficking. Gene augmentation is a promising therapeutic strategy, and there are several...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 37894906
DOI: 10.3390/ijms242015225
PMCID: PMC10607001

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