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Scientific Reports|October 1, 2017
Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease modelJamal Alzubi, Celeste Pallant, Claudio Mussolino, et al.
Journal of Molecular Biology|March 3, 2009
The functional role of S/MARs in episomal vectors as defined by the stress-induced destabilization profile of the vector sequencesAristeidis Giannakopoulos, Eleana F Stavrou, Ioannis Zarkadis, et al.
Blood|March 2, 2006
Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A miceCarol H Miao, Peiqing Ye, Arthur R Thompson, et al.
Proceedings of the National Academy of Sciences of the United States of America|December 8, 2010
Impaired induction of DNA lesions during immunoglobulin class-switch recombination in humans influences end-joining repairSven Kracker, Kohsuke Imai, Pauline Gardès, et al.
Blood|September 14, 2012
Excess F-actin mechanically impedes mitosis leading to cytokinesis failure in X-linked neutropenia by exceeding Aurora B kinase error correction capacityDale A Moulding, Emad Moeendarbary, Leo Valon, et al.
European Journal of Haematology|March 24, 2007
Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cellsElena K Siapati, Brian W Bigger, Karl Kashofer, et al.
Human Gene Therapy|November 28, 2019
Differential Transgene Silencing of Myeloid-Specific Promoters in the <i>AAVS1</i> Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid CellsDenise Klatt, Erica Cheng, Dirk Hoffmann, et al.
Molecular Therapy. Methods & Clinical Development|March 23, 2023
An improved medium formulation for efficient <i>ex vivo</i> gene editing, expansion and engraftment of hematopoietic stem and progenitor cellsRajeev Rai, Asma Naseem, Winston Vetharoy, et al.
Stem Cells (Dayton, Ohio)|November 8, 2012
Brief report: self-organizing neuroepithelium from human pluripotent stem cells facilitates derivation of photoreceptorsCédric Boucherie, Sayandip Mukherjee, Els Henckaerts, et al.
Plos One|March 17, 2011
Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cellsSayandip Mukherjee, Giorgia Santilli, Michael P Blundell, et al.
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