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Blood|March 17, 2009
Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfectionRobert E Throm, Annastasia A Ouma, Sheng Zhou, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 1, 2019
AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular MyopathyJean-Baptiste Dupont, Jianjun Guo, Edith Renaud-Gabardos, et al.
Human Gene Therapy|March 18, 2011
Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trialJames A Allay, Susan Sleep, Scott Long, et al.
Blood|May 12, 2010
A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cellsSheng Zhou, Disha Mody, Suk See DeRavin, et al.
Journal of Pediatric Surgery|January 9, 2007
Intravascular administration of tumor tropic neural progenitor cells permits targeted delivery of interferon-beta and restricts tumor growth in a murine model of disseminated neuroblastomaPaxton V Dickson, John B Hamner, Rebecca A Burger, et al.
Blood|December 3, 2005
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liverAmit C Nathwani, John T Gray, Catherine Y C Ng, et al.
Molecular Therapy. Methods & Clinical Development|February 25, 2022
Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector prepsMark A Brimble, Pei-Hsin Cheng, Stephen M Winston, et al.
Molecular Therapy. Advances|May 15, 2026
Rational engineering of the P5 TRS-mimic site and REP78/68 start codon yields promoter variants that improve rAAV purity while maintaining high titersStephen M Winston, Mark A Brimble, Kristin B Wiggins, et al.
Blood|December 3, 2011
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhageChristopher Binny, Jenny McIntosh, Marco Della Peruta, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 20, 2011
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteinsAmit C Nathwani, Cecilia Rosales, Jenny McIntosh, et al.
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