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Lending a helping hand, screening chemical libraries for compounds that enhance β-hexosaminidase A activity in GM2 gangliosidosis cells

Enzyme enhancement therapy is an emerging therapeutic approach that has the potential to treat many genetic diseases. Candidate diseases are those associated with a mutant protein that has difficulty folding and/or assembling into active oligomers in the endoplasmic reticulum. Many lysosomal storage...

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Detalhes bibliográficos
Main Authors: Tropak, Michael B., Mahuran, Don
Formato: Artigo
Idioma:Inglês
Publicado em: 2007
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2910757/
https://ncbi.nlm.nih.gov/pubmed/17894780
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/j.1742-4658.2007.06040.x
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