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Molecular Therapy. Methods & Clinical Development|May 1, 2018
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined ImmunodeficiencyValentina Poletti, Sabine Charrier, Guillaume Corre, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 19, 2007
Stable gene transfer to muscle using non-integrating lentiviral vectorsLuis Apolonia, Simon N Waddington, Carolina Fernandes, et al.
Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|January 17, 2013
Polyphenon [corrected] E enhances the antitumor immune response in neuroblastoma by inactivating myeloid suppressor cellsGiorgia Santilli, Izabela Piotrowska, Sandra Cantilena, et al.
Molecular Therapy. Nucleic Acids|February 22, 2024
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAVSriram Vaidyanathan, Jenny L Kerschner, Alekh Paranjapye, et al.
Blood|April 26, 2007
Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cellsFang Zhang, Susannah I Thornhill, Steven J Howe, et al.
The Journal of Investigative Dermatology|January 11, 2013
T-cell reconstitution after thymus xenotransplantation induces hair depigmentation and lossAnna L Furmanski, Ryan F L O'Shaughnessy, Jose Ignacio Saldana, et al.
Elife|November 2, 2020
Wiskott Aldrich syndrome protein regulates non-selective autophagy and mitochondrial homeostasis in human myeloid cellsElizabeth Rivers, Rajeev Rai, Jonas Lötscher, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 6, 2004
Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A micePeiqing Ye, Arthur R Thompson, Rita Sarkar, et al.
Blood|March 21, 2012
Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndromeAlexander Astrakhan, Blythe D Sather, Byoung Y Ryu, et al.
Blood|February 24, 2016
Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndromeNicholas Hubbard, David Hagin, Karen Sommer, et al.
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