Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency

Por Mihelec, Marija, Pearson, Rachael A., Robbie, Scott J., Buch, Prateek K., Azam, Selina A., Bainbridge, James W.B., Smith, Alexander J., Ali, Robin R.
Publicado em 2011

Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179

Por Nishiguchi, Koji M., Carvalho, Livia S., Rizzi, Matteo, Powell, Kate, Holthaus, Sophia-Martha kleine, Azam, Selina A., Duran, Yanai, Ribeiro, Joana, Luhmann, Ulrich F. O., Bainbridge, James W. B., Smith, Alexander J., Ali, Robin R.
Publicado no Nat Commun (2015)