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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.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 17, 2014
Nonallele specific silencing of ataxin-7 improves disease phenotypes in a mouse model of SCA7Pavitra S Ramachandran, Ryan L Boudreau, Kellie A Schaefer, et al.Cell Chemical Biology|September 22, 2023
Boosting glycolysis to combat fragile bone in type 1 diabetesZixue Jin, Brendan LeeTrends in Molecular Medicine|December 10, 2002
Urea-cycle disorders as a paradigm for inborn errors of hepatocyte metabolismAsad Mian, Brendan LeePageof 46