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Improved Retroviral Vector Design Results in Sustained Expression after Adult Gene Therapy in Mucopolysaccharidosis I Mice

BACKGROUND: Mucopolysaccharidosis I (MPS I) is a lysosomal storage disease due to α-L-iduronidase (IDUA) deficiency that results in the accumulation of glycosaminoglycans (GAG). Gene therapy can reduce most clinical manifestations, but mice that receive transfer as adults lose expression unless they...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Herati, Ramin Sedaghat, Ma, Xiucui, Tittiger, Mindy, Ohlemiller, Kevin K, Kovacs, Attila, Ponder, Katherine P.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2008
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC2829257/
https://ncbi.nlm.nih.gov/pubmed/18613275
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jgm.1229
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