Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys

Many neuropathic diseases cause early, irreversible neurologic deterioration, which warrants therapeutic intervention during the first months of life. In the case of mucopolysaccharidosis type I, a recessive lysosomal storage disorder that results from a deficiency of the lysosomal enzyme α-l-iduron...

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Dettagli Bibliografici
Pubblicato in:Hum Gene Ther
Autori principali: Hordeaux, Juliette, Hinderer, Christian, Buza, Elizabeth L., Louboutin, Jean-Pierre, Jahan, Tahsin, Bell, Peter, Chichester, Jessica A., Tarantal, Alice F., Wilson, James M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc., publishers 2019
Soggetti:
Research Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6703245/
https://ncbi.nlm.nih.gov/pubmed/31017018
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.012
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