AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in a mouse model of familial dysautonomia.
Anil K. Chekuri, Krishnakanth Kondabolu, Emily G. Kirchner…·Susan A. Slaugenhaupt
This study uses AAV2-delivered exon-specific U1 snRNA to correct ELP1 splicing and rescue optic neuropathy in a FD mouse model.