Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa
<p>X-linked retinitis pigmentosa (XLRP) is generally a severe form of retinitis pigmentosa, a neurodegenerative, blinding disorder of the retina. 70% of XLRP cases are due to mutations in the retina-specific isoform of the gene encoding retinitis pigmentosa GTPase regulator (<em>...
| मुख्य लेखकों: | , , , , , , , , |
|---|---|
| स्वरूप: | Journal article |
| भाषा: | English |
| प्रकाशित: |
Cell Press
2017
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