Showing results (11-20 of 21) with videos related to
Sort By:
Pageof 3
American Journal of Human Genetics|May 31, 2016
RNA Interference Prevents Autosomal-Dominant Hearing LossSeiji B Shibata, Paul T Ranum, Hideaki Moteki, et al.Molecular Therapy. Nucleic Acids|August 7, 2023
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSFEllie M Carrell, Yong Hong Chen, Paul T Ranum, et al.Science Translational Medicine|May 14, 2025
An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and miceLuis Tecedor, Yong Hong Chen, David E Leib, et al.Biorxiv : the Preprint Server for Biology|August 30, 2023
APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer diseaseRosemary J Jackson, Megan S Keiser, Jonah C Meltzer, et al.Nature Medicine|October 19, 2021
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brainMegan S Keiser, Paul T Ranum, Carolyn M Yrigollen, et al.Nature Communications|May 6, 2023
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switchingJennine M Dawicki-McKenna, Alex J Felix, Elisa A Waxman, et al.Plos Genetics|March 28, 2015
HOMER2, a stereociliary scaffolding protein, is essential for normal hearing in humans and miceHela Azaiez, Amanda R Decker, Kevin T Booth, et al.Human Molecular Genetics|July 23, 2021
Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeuticsSergey S Akimov, Mali Jiang, Amanda J Kedaigle, et al.Human Genetics|March 13, 2016
Comprehensive genetic testing in the clinical evaluation of 1119 patients with hearing lossChristina M Sloan-Heggen, Amanda O Bierer, A Eliot Shearer, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 20, 2024
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer diseaseRosemary J Jackson, Megan S Keiser, Jonah C Meltzer, et al.Pageof 3