AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression.
2017
Gicquel, E., et al. (2017).
Hum Mol Genet 26(10): 1952-1965.
2017
Gicquel, E., et al. (2017).
Hum Mol Genet 26(10): 1952-1965.