Gene dosage manipulation to alleviate manifestations of hereditary PAX6 haploinsufficiency
Gene dosage manipulation to alleviate manifestations of hereditary PAX6 haploinsufficiency
Rabiee B, Anwar K, Shen X, Putra I, Liu M, Jung R, Afsharkhamseh A, Rosenblatt M, Fishman G, Liu X, et al. Gene dosage manipulation to alleviate manifestations of hereditary PAX6 haploinsufficiency. Science Translational Medicine (accepted). 2020.
