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Frontiers in Genome Editing|June 9, 2022
Genome Editing With TALEN, CRISPR-Cas9 and CRISPR-Cas12a in Combination With AAV6 Homology Donor Restores T Cell Function for XLPBenjamin C Houghton, Neelam Panchal, Simone A Haas, et al.
Blood|August 9, 2008
Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasisAlmut Meyer-Bahlburg, Shirly Becker-Herman, Stephanie Humblet-Baron, et al.
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.
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.
Stem Cell Reports|September 24, 2019
Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous PseudogenesDenise Klatt, Erica Cheng, Friederike Philipp, et al.
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