Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa

Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa

<p>Dominant mutations in RHO (rhodopsin) are the most common cause of autosomal dominant retinitis pigmentosa (RHO-adRP). RHO-adRP causes progressive loss of rod cells, followed by cone cells, leading to blindness. This disease is a candidate for CRISPR gene editing, as reduction of mutant rho...

Полное описание

Библиографические подробности
Главный автор:	Peddle, CF
Другие авторы:	MacLaren, RE
Формат:	Диссертация
Язык:	English
Опубликовано:	2021
Предметы:	Retinal degeneration Gene therapy CRISPR (Genetics) Retinitis pigmentosa

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