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...

Bibliografski detalji
Glavni autor:	Peddle, CF
Daljnji autori:	MacLaren, RE
Format:	Disertacija
Jezik:	English
Izdano:	2021
Teme:	Retinal degeneration Gene therapy CRISPR (Genetics) Retinitis pigmentosa

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