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Molecular Therapy : the Journal of the American Society of Gene Therapy|March 29, 2024
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter diseaseJessica A Herstine, Pi-Kai Chang, Sergiy Chornyy, et al.
ASN Neuro|April 16, 2015
AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous systemHannah E Rockwell, Victoria J McCurdy, Samuel C Eaton, et al.
Journal of Clinical Immunology|February 10, 2015
Spectrum and management of complement immunodeficiencies (excluding hereditary angioedema) across EuropeA J Turley, B Gathmann, C Bangs, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 19, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disordersBeverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, et al.
Immunotargets and Therapy|December 9, 2020
Development of Anti-Yersinia pestis Human Antibodies with Features Required for Diagnostic and Therapeutic ApplicationsAntonietta M Lillo, Nileena Velappan, Julia M Kelliher, et al.
Human Gene Therapy|January 31, 2017
Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate BrainDiane Golebiowski, Imramsjah M J van der Bom, Churl-Su Kwon, et al.
Elife|August 14, 2024
Antibody characterization is critical to enhance reproducibility in biomedical researchRichard A Kahn, Harvinder Virk, Carl Laflamme, et al.
Human Gene Therapy|January 11, 2018
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)Allison M Bradbury, Mohammed A Rafi, Jessica H Bagel, et al.
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