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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Autori principali:	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
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2025-06-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: Oligonucleotides: Therapies and Applications late-onset Pompe disease lysosomal storage disease oligonucleotide therapeutics RNA medicines splice switching antisense oligonucleotide
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2162253125000782
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