Genome Editing as a Treatment for the Most Prevalent Causative Genes of Autosomal Dominant Retinitis Pigmentosa

Genome Editing as a Treatment for the Most Prevalent Causative Genes of Autosomal Dominant Retinitis Pigmentosa

Inherited retinal dystrophies (IRDs) are a clinically and genetically heterogeneous group of diseases with more than 250 causative genes. The most common form is retinitis pigmentosa. IRDs lead to vision impairment for which there is no universal cure. Encouragingly, a first gene supplementation the...

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Bibliographic Details
Main Authors: Michalitsa Diakatou, Gaël Manes, Beatrice Bocquet, Isabelle Meunier, Vasiliki Kalatzis
Format: Article
Language:English
Published: MDPI AG 2019-05-01
Series:International Journal of Molecular Sciences
Subjects:
Inherited retinal dystrophies
autosomal dominant retinitis pigmentosa
photoreceptors
loss-of-function
gain-of-function
dominant-negative
CRISPR/Cas
gene supplementation
genome-editing
AAV vector
Online Access:https://www.mdpi.com/1422-0067/20/10/2542

Internet

https://www.mdpi.com/1422-0067/20/10/2542

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