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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...

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Main Authors: Herati, Ramin Sedaghat, Ma, Xiucui, Tittiger, Mindy, Ohlemiller, Kevin K, Kovacs, Attila, Ponder, Katherine P.
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2008
Ábhair:
Rochtain Ar Líne: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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