Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model
Blindness in Bardet-Biedl syndrome (BBS) is caused by dysfunction and loss of photoreceptor cells in the retina. BBS10, mutations of which account for approximately 21% of all BBS cases, encodes a chaperonin protein indispensable for the assembly of the BBSome, a cargo adaptor important for ciliary...
Main Authors: | , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2023-03-01
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Series: | Molecular Therapy: Nucleic Acids |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2162253122003286 |