Antisense oligonucleotides targeting valosin‐containing protein ameliorate muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy
Year: 2025
Type: article
Language: en
Topic: Muscle Physiology and Disorders
Authors and affiliations:
raw_author_name: Pallabi Pal
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Michele Carrer
raw_affiliation_strings: Ionis Pharmaceuticals, Inc.  Carlsbad California USA
Ionis Pharmaceuticals, Inc., Carlsbad, California, USA
raw_author_name: Lan Weiss
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Olga G. Jaime
raw_affiliation_strings: Department of Physiology and Biophysics School of Medicine University of California  Irvine California USA
Sue and Bill Gross Stem Cell Research Center University of California  Irvine California USA
Department of Physiology and Biophysics, School of Medicine, University of California, Irvine, California, USA
raw_author_name: Cheng Cheng
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Alyaa Shmara
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Victoria Boock
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Danae Bosch
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Marwan Youssef
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Yasamin Fazeli
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA
raw_author_name: Megan Afetian
raw_affiliation_strings: Ionis Pharmaceuticals, Inc.  Carlsbad California USA
Ionis Pharmaceuticals, Inc., Carlsbad, California, USA
raw_author_name: Tamar R. Grossman
raw_affiliation_strings: Ionis Pharmaceuticals, Inc.  Carlsbad California USA
Ionis Pharmaceuticals, Inc., Carlsbad, California, USA
raw_author_name: Michael R. Hicks
raw_affiliation_strings: Department of Physiology and Biophysics School of Medicine University of California  Irvine California USA
Sue and Bill Gross Stem Cell Research Center University of California  Irvine California USA
Department of Physiology and Biophysics, School of Medicine, University of California, Irvine, California, USA
raw_author_name: Paymaan Jafar‐nejad
raw_affiliation_strings: Ionis Pharmaceuticals, Inc.  Carlsbad California USA
Ionis Pharmaceuticals, Inc., Carlsbad, California, USA
raw_author_name: Virginia Kimonis
raw_affiliation_strings: Department of Pediatrics Division of Genetics and Genomic Medicine University of California  Irvine California USA
Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California, Irvine, California, USA