CRISPR/Cas9-Mediated Excision of ALS/FTD-Causing Hexanucleotide Repeat Expansion in <i>C9ORF72</i> rescues major disease mechanisms <i>in vivo</i> and <i>in vitro</i>
Katharina E. Meijboom(University of Massachusetts Chan Medical School), Christian Mueller(AbbVie (Germany))
Cited by 1
Related Papers
MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression
|Molecular Therapy|2010|168
Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression
|Journal of Clinical Investigation|2013|160