Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

Nicole McNeer(Yale University), Erica Schleifman(Yale University), Amy Cuthbert(University of Massachusetts Chan Medical School), Michael A. Brehm(University of Massachusetts Chan Medical School), Andrew Jackson(Yale University), Christopher H.K. Cheng(Yale University), Kavi Anandalingam(Yale University), Priti Kumar(Yale University), Leonard D. Shultz(Jackson Laboratory), Dale L. Greiner(University of Massachusetts Chan Medical School), W. Mark Saltzman(Yale University), Peter M. Glazer(Yale University)
Gene Therapy
October 18, 2012
Cited by 77

Abstract


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