Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse

Duchenne muscular dystrophy (DMD) is caused by mutations in DMD, resulting in loss of dystrophin, which is essential to muscle health. DMD “exon skipping” uses anti-sense oligo-nucleotides (AONs) to force specific exon exclusion during mRNA processing to restore reading frame and rescue of partially...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Wang, Derek W., Mokhonova, Ekaterina I., Kendall, Genevieve C., Becerra, Diana, Naeini, Yalda B., Cantor, Rita M., Spencer, Melissa J., Nelson, Stanley F., Miceli, M. Carrie
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
Témata:
Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5992346/
https://ncbi.nlm.nih.gov/pubmed/29858053
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.02.002
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky