A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy
Titel:
A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy
Auteur:
Dhoke, Neha R. Kim, Hyunkee Selvaraj, Sridhar Azzag, Karim Zhou, Haowen Oliveira, Nelio A.J. Tungtur, Sudheer Ortiz-Cordero, Carolina Kiley, James Lu, Qi Long Bang, Anne G. Perlingeiro, Rita C.R.