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

Autor:	Hans R. Schöler, Joon M. Pijnenburg, T. van Gestel, W. Pijnappel, E. van der Wal, Atze J. Bergsma, A.T. van der Ploeg, Marcos J. Araúzo-Bravo, S. in ‘t Groen, Holm Zaehres
Rok vydání:	2017
Předmět:	0301 basic medicine Skeletal muscle Biology Molecular biology 03 medical and health sciences Exon 030104 developmental biology medicine.anatomical_structure Neurology Pediatrics Perinatology and Child Health Antisense oligonucleotides RNA splicing medicine Neurology (clinical) Genetics (clinical)
Zdroj:	Neuromuscular Disorders. 27:S161
ISSN:	0960-8966
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::71a25594bcb3c74e68ef1fd6cc7da998 https://doi.org/10.1016/j.nmd.2017.06.248 Zobrazit plný text záznamu Full Text from ScienceDirect