Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa
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Publisher
Springer International Publishing
Link
http://link.springer.com/content/pdf/10.1007/978-3-030-27378-1_19
Reference22 articles.
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3. Burnight ER, Gupta M, Wiley LA et al (2017) Using CRISPR-Cas9 to generate gene-corrected autologous iPSCs for the treatment of inherited retinal degeneration. Mol Ther 25:1999–2013
4. Chadderton N, Millington-Ward S, Palfi A et al (2009) Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy. Mol Ther 17:593–599
5. Cideciyan AV, Sudharsan R, Dufour VL et al (2018) Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector. Proc Natl Acad Sci U S A 115:E8547–E8556
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