Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Mark Bigg"'
Autor:
Kevin K. Ohlemiller, Attila Kovacs, Li He, Douglas M. Tollefsen, Jason A. Metcalf, Axel Schambach, Xiucui Ma, Susan Wu, Bruce Linders, Katherine P. Ponder, Mark Bigg
Publikováno v:
Molecular Therapy. 18(2):334-342
Mucopolysaccharidosis I (MPS I) is a lysosomal storage disease due to deficiency in alpha-L-iduronidase (IDUA) that results in accumulation of glycosaminoglycans (GAGs) throughout the body, causing numerous clinical defects. Intravenous administratio
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35ac421f9ac25c539b04f9ac7d30f166
Autor:
Katherine P. Ponder, Attila Kovacs, Mindy Tittiger, Sarah Popelka, Yuli Liu, Mark Bigg, Anne K. Hennig, Baomei Wang, Ramin S. Herati, Xiucui Ma
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 15(5)
Mucopolysaccharidosis I (MPS I) is caused by deficient alpha-L-iduronidase (IDUA) activity and results in the accumulation of glycosaminoglycans and multisystemic disease. Gene therapy could program cells to secrete mannose 6-phosphate-modified IDUA,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4b13ac0e62af6a6df7337df2907b54f6
https://pubmed.ncbi.nlm.nih.gov/17311010
https://pubmed.ncbi.nlm.nih.gov/17311010
Autor:
Katherine P. Ponder, Sarah Popelka, Mark Bigg, Anne K. Hennig, Attila Kovacs, Mindy Tittiger, Yuli Liu, Ramin S. Herati, Xiucui Ma
Publikováno v:
Molecular Genetics and Metabolism. 92:34
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::00183e9210e6941aa36a08548a566b82
https://doi.org/10.1016/j.ymgme.2007.08.114
https://doi.org/10.1016/j.ymgme.2007.08.114