Lentiviral vectors can be used for full-length dystrophin gene therapy
Sign Up to like & getrecommendations! Published in 2017 at "Scientific Reports"
DOI: 10.1038/s41598-017-00152-5
Abstract: Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient muscle fibres. Current DMD gene therapy strategies rely on the expression of internally deleted forms of dystrophin, missing important functional domains.… read more here.
Keywords: length dystrophin; gene therapy; full length; lentiviral vectors ... See more keywords
Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy
Sign Up to like & getrecommendations! Published in 2017 at "Scientific Reports"
DOI: 10.1038/srep46880
Abstract: This corrects the article DOI: 10.1038/srep44775. read more here.
Keywords: vectors used; full length; erratum lentiviral; lentiviral vectors ... See more keywords
Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes
Sign Up to like & getrecommendations! Published in 2022 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms23169176
Abstract: Duchenne muscular dystrophy (DMD) is the most common fatal muscle disease, with an estimated incidence of 1/3500–1/5000 male births, and it is associated with mutations in the X-linked DMD gene encoding dystrophin, the largest known… read more here.
Keywords: full length; addition; length dystrophin; dmd ... See more keywords