Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.
Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, Jacobson SG, Hauswirth WW.
Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):15112-7. Epub 2008 Sep 22.PMID: 18809924 [PubMed - indexed for MEDLINE]Related articlesFree article