2016
DOI: 10.1016/s1525-0016(16)32947-1
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138. Potential Therapeutic Treatment of Friedrich's Ataxia Using Highly Specific Engineered Meganucleases

Abstract: Mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene cause severe X-linked retinitis pigmentosa (XLRP). More than 80% of the mutations are located in the terminal exon ORF15 of the RPGR gene. Genome editing, which represents a novel approach to treat monogenic disorders, is based on highly specific nucleases that cleave or nick at a chosen position within the complex genome followed by the repair of the double or single strand break (DSB or SSB) by endogenous repair mechanisms. The major pathways… Show more

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