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Molecular Therapy : the Journal of the American Society of Gene Therapy|April 4, 2025
Current trends in gene therapy to treat inherited disorders of the brainZaneta Matuszek, Brandon L Brown, Carolyn M Yrigollen, et al.
Neurobiology of Disease|April 17, 2018
Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivoMark L Schultz, Luis Tecedor, Elena Lysenko, et al.
Genome Biology|April 28, 2022
DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencingLi Fang, Qian Liu, Alex Mas Monteys, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 18, 2009
Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimer's diseaseEdgardo Rodríguez-Lebrón, Cynthia M Gouvion, Steven A Moore, et al.
Molecular Therapy. Nucleic Acids|July 9, 2024
Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelityJesse A Weber, Jonathan F Lang, Ellie M Carrell, et al.
Nature Communications|August 1, 2019
Standard screening methods underreport AAV-mediated transduction and gene editingJonathan F Lang, Sushila A Toulmin, Kasey L Brida, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|November 15, 2013
CLN3 loss disturbs membrane microdomain properties and protein transport in brain endothelial cellsLuis Tecedor, Colleen S Stein, Mark L Schultz, et al.
Molecular Therapy. Nucleic Acids|January 25, 2013
Manipulation of Cell Physiology Enables Gene Silencing in Well-differentiated Airway EpitheliaSateesh Krishnamurthy, Mark A Behlke, Shyam Ramachandran, et al.
Molecular Therapy. Nucleic Acids|October 15, 2014
Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In VivoJames C Geoghegan, Nicholas W Keiser, Anna Okulist, et al.
Plos Genetics|October 9, 2008
Diverse splicing patterns of exonized Alu elements in human tissuesLan Lin, Shihao Shen, Anne Tye, et al.
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