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Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.
- Published in:
- Human Molecular Genetics, 2010, v. 19, n. 4, p. 735, doi. 10.1093/hmg/ddp540
- By:
- Publication type:
- Article
Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.
- Published in:
- Human Molecular Genetics, 2009, v. 18, n. 12, p. 2099, doi. 10.1093/hmg/ddp133
- By:
- Publication type:
- Article
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
- Published in:
- 2018
- By:
- Publication type:
- journal article
Cover Image, Volume 69, Issue 9.
- Published in:
- Glia, 2021, v. 69, n. 9, p. C1, doi. 10.1002/glia.24057
- By:
- Publication type:
- Article
RNAi‐mediated suppression of vimentin or glial fibrillary acidic protein prevents the establishment of Müller glial cell hypertrophy in progressive retinal degeneration.
- Published in:
- Glia, 2021, v. 69, n. 9, p. 2272, doi. 10.1002/glia.24034
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- Publication type:
- Article
Nanotube‐like processes facilitate material transfer between photoreceptors.
- Published in:
- EMBO Reports, 2021, v. 22, n. 11, p. 1, doi. 10.15252/embr.202153732
- By:
- Publication type:
- Article
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
- Published in:
- Human Molecular Genetics, 2019, v. 28, n. 23, p. 3867, doi. 10.1093/hmg/ddz210
- By:
- Publication type:
- Article