Associated Papers
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Harmatz P et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2022
The dual-function disabled-1 PTB domain exhibits site independence in binding phosphoinositide and peptide ligands.
Stolt PC et al.
Biochemistry, 2004
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