Targeted gene addition in human CD34 super(+) hematopoietic cells for correction of X-linked chronic...
Targeted gene addition in human CD34 super(+) hematopoietic cells for correction of X-linked chronic granulomatous disease
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Author / Creator
De Ravin, Suk See , Reik, Andreas , Liu, Pei-Qi , Li, Linhong , Wu, Xiaolin , Su, Ling , Raley, Castle , Theobald, Narda , Choi, Uimook , Song, Alexander H , Chan, Andy , Pearl, Jocelynn R , Paschon, David E , Lee, Janet , Newcombe, Hannah , Koontz, Sherry , Sweeney, Colin , Shivak, David A , Zarember, Kol A , Peshwa, Madhusudan V , Gregory, Philip D , Urnov, Fyodor D and Malech, Harry L
Journal title
Language
English
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Contents
Gene therapy with genetically modified human CD34 super(+) hematopoietic stem and progenitor cells (HSPCs) may be safer using targeted integration (TI) of transgenes into a genomic 'safe harbor' site rather than random viral integration. We demonstrate that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-ass...
Alternative Titles
Full title
Targeted gene addition in human CD34 super(+) hematopoietic cells for correction of X-linked chronic granulomatous disease
Authors, Artists and Contributors
Author / Creator
Reik, Andreas
Liu, Pei-Qi
Li, Linhong
Wu, Xiaolin
Su, Ling
Raley, Castle
Theobald, Narda
Choi, Uimook
Song, Alexander H
Chan, Andy
Pearl, Jocelynn R
Paschon, David E
Lee, Janet
Newcombe, Hannah
Koontz, Sherry
Sweeney, Colin
Shivak, David A
Zarember, Kol A
Peshwa, Madhusudan V
Gregory, Philip D
Urnov, Fyodor D
Malech, Harry L
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Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_1785229748
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1785229748
Other Identifiers
ISSN
1087-0156
DOI
10.1038/nbt.3513
