Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.
Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice.
Schmid RS, Deng X, Panikker P, Msackyi M, Breton C, Wilson JM
The Journal Of Clinical Investigation