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2/12/2020
A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency
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Science Advances
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6/27/2019
CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice.
Wang L, Yang Y, Breton CA, White J, Zhang J, Che Y, Saveliev A, McMenamin D, He Z, Latshaw C, Li M, Wilson JM
Blood
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3/01/2016
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM
Nature Biotechnology
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5/01/1998
Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers.
Jooss K, Yang Y, Fisher KJ, Wilson JM
Journal Of Virology
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10/15/1997
Amelioration of collagen-induced arthritis by CD95 (Apo-1/Fas)-ligand gene transfer.
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The Journal Of Clinical Investigation
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Recombinant adeno-associated virus for muscle directed gene therapy.
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Nature Medicine