Work

Antisense oligonucleotides promote exon inclusion and correct the common c.-32-13T > G (IVS1) GAA splicing variant in iPS-derived skeletal muscle cells from Pompe patients

Year: 2017

Type: article

Source: Neuromuscular Disorders

DOI: 10.1016/j.nmd.2017.06.248

Authors: W. Pijnappel, E. van der Wal, A. Bergsma, T. van Gestel, J. Pijnenburg, S. In ‘t Groen, H. Zaehres, M. Araúzo-Bravo, H. Schöler, A. van der Ploeg

Institutions: Erasmus MC, Max Planck Institute for Molecular Biomedicine

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2755215583

FWCI:

Cites:

Cited by: 1

Related to: 10