Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease

Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease

Late-onset Pompe disease (LOPD) is overwhelmingly caused by a single mutation that disrupts splicing of acid-alpha glucosidase (GAA) and results in the accumulation of lysosomal glycogen in muscle cells leading to progressive muscle weakness in patients. Current therapeutics for LOPD do not meet the...

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Main Authors: Ryan A. Oliver, Meghan E. Ahern, Perla G. Castaneda, Tushare Jinadasa, Anirban Bardhan, Kathy Y. Morgan, Kristin Ha, Kailash Adhikari, Nino Jungels, Noa Liberman, Anindita Mitra, Christopher D. Greer, Alec M. Wright, Emily G. Thompson, Stephanie Garcia, Elena Copson, Senkara Allu, Xuyu Tan, Alex J. Callahan, Bao Zhong Cai, Vincent Guerlavais, Kevin J. Kim, Annika B. Malmberg
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
late-onset Pompe disease
lysosomal storage disease
oligonucleotide therapeutics
RNA medicines
splice switching antisense oligonucleotide
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125000782
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