A large animal model of Spinal Muscular Atrophy and correction of phenotype

OBJECTIVES: Spinal muscular atrophy (SMA) is caused by reduced levels of SMN which results in motoneuron loss. Therapeutic strategies to increase SMN levels including drug compounds, antisense oligonucleotides or scAAV9 gene therapy have proved effective in mice. We wished to determine whether reduc...

Cijeli opis

Spremljeno u:
Bibliografski detalji
Izdano u:Ann Neurol
Glavni autori: Duque, Sandra I., Arnold, W. David, Odermatt, Philipp, Li, Xiaohui, Porensky, Paul N., Schmelzer, Leah, Meyer, Kathrin, Kolb, Stephen J., Schümperli, Daniel, Kaspar, Brian K., Burghes, Arthur H. M.
Format: Artigo
Jezik:Inglês
Izdano: 2015
Teme:
Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4453930/
https://ncbi.nlm.nih.gov/pubmed/25516063
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24332
Oznake: Dodaj oznaku
Bez oznaka, Budi prvi tko označuje ovaj zapis!

Similar Items