Dual-targeting snRNA gene therapy rescues STMN2 and UNC13A splicing in TDP-43 proteinopathies
Year: 2025
Type: article
Language: en
Topic: Amyotrophic Lateral Sclerosis Research
Authors and affiliations:
raw_author_name: Trent A. Gomberg
raw_affiliation_strings: Biomedical Sciences Graduate Program, University of California San Diego, La Jolla, CA, USA
Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA
Sanford Stem Cell Institute Innovation Center and Sanford Consortium for Regenerative Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Sara Elmsaouri
raw_affiliation_strings: Biomedical Sciences Graduate Program, University of California San Diego, La Jolla, CA, USA
Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA
Sanford Stem Cell Institute Innovation Center and Sanford Consortium for Regenerative Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Hema M. Kopalle
raw_affiliation_strings: Biological Sciences Graduate Program, University of California San Diego, La Jolla, CA, USA
Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA
Sanford Stem Cell Institute Innovation Center and Sanford Consortium for Regenerative Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Michael W. Baughn
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Melinda S. Beccari
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Hoogenraad Lab, Genentech, South San Francisco, CA, USA
raw_author_name: Melissa McAlonis-Downes
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Jonathan W. Artates
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Deepak Pant
raw_affiliation_strings: Biological Sciences Graduate Program, University of California San Diego, La Jolla, CA, USA
Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA
Sanford Stem Cell Institute Innovation Center and Sanford Consortium for Regenerative Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Harriet Mak
raw_affiliation_strings: Capstan Therapeutics, San Diego, CA, USA
raw_author_name: Aaron A. Smargon
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA
Sanford Stem Cell Institute Innovation Center and Sanford Consortium for Regenerative Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Tynan C. Sander
raw_author_name: Esaul Garcia
raw_affiliation_strings: Chemical Engineering Department, University of California San Diego, La Jolla, CA, USA
raw_author_name: Dominic P. Lee
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA
Sanford Stem Cell Institute Innovation Center and Sanford Consortium for Regenerative Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Don W. Cleveland
raw_affiliation_strings: Department of Cellular & Molecular Medicine, University of California San Diego, La Jolla, CA, USA
raw_author_name: Gene W. Yeo