S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA.
Koirala A, Makkia RS, Conley SM, Cooper MJ, Naash MI. S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA. Hum Mol Genet. 2013 Apr 15; 22(8):1632-42.