Zobrazeno 1 - 6
of 6
pro vyhledávání: '"Shobha Ramsubir"'
Publikováno v:
Molecular Therapy. 15:1174-1181
Gene therapy for Fabry disease, a deficiency in alpha-galactosidase A (alpha-gal A) activity, has the potential to provide a cure for the disorder with a single treatment. Despite modifications to existing vectors, concerns have arisen regarding the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8d3f8b05f272cf1b18679941e717023d
https://doi.org/10.1038/sj.mt.6300147
https://doi.org/10.1038/sj.mt.6300147
Autor:
Vanessa I. Rasaiah, Christopher Siatskas, Roscoe O. Brady, Shobha Ramsubir, Makoto Yoshimitsu, Gary J. Murray, Takahiro Nonaka, Jeffrey A. Medin, Koji Higuchi, S-B Liang
Publikováno v:
Gene Therapy. 14:256-265
A deficiency in alpha-galactosidase A (alpha-gal A) activity causes Fabry disease. Virus-based delivery of genes can correct cells and establish a sustained supply of therapeutic proteins. Recombinant lentiviral vectors (LVs) show promise in this con
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4d8f0900aad00b279e2e3c2ac9198149
https://doi.org/10.1038/sj.gt.3302839
https://doi.org/10.1038/sj.gt.3302839
Autor:
Thierry Levade, Jeffrey A. Medin, Stéphane Carpentier, Takahiro Nonaka, Carmen Bedia Girbés, Shobha Ramsubir
Publikováno v:
Molecular Genetics and Metabolism
Molecular Genetics and Metabolism, 2008, 95 (3), pp.133-41. ⟨10.1016/j.ymgme.2008.08.003⟩
Molecular Genetics and Metabolism, 2008, 95 (3), pp.133-41. ⟨10.1016/j.ymgme.2008.08.003⟩
International audience; Farber disease is a rare lysosomal storage disorder (LSD) caused by a deficiency of acid ceramidase (AC) activity and subsequent accumulation of ceramide. Currently, there is no treatment for Farber disease beyond palliative c
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::228796f388f27b90cf2b7fc9ff37bc97
https://www.hal.inserm.fr/inserm-00409375
https://www.hal.inserm.fr/inserm-00409375
Publikováno v:
Experimental biology and medicine (Maywood, N.J.). 231(5)
Choline is an essential nutrient for all cells because it plays a role in the synthesis of the membrane phospholipid components of the cell membranes, as a methyl-group donor in methionine metabolism as well as in the synthesis of the neurotransmitte
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::776a4178588dc9ee2bcafcf71b438575
https://pubmed.ncbi.nlm.nih.gov/16636297
https://pubmed.ncbi.nlm.nih.gov/16636297
Publikováno v:
Molecular Therapy. 9:S330
Farber disease is a rare, lysosomal storage disorder that is caused by the deficient activity of the enzyme acid ceramidase. As a result, the sphingolipid ceramide accumulates in the lysosomal compartment. Patients typically present with subcutaneous
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::887fc6df0fcce30b7ec7df1618b092ef
https://doi.org/10.1016/j.ymthe.2004.06.775
https://doi.org/10.1016/j.ymthe.2004.06.775
Publikováno v:
Journal of Cardiology. (2):245-254
SummaryFor some applications, the success of gene therapy depends on the efficiency of gene transfer into target organs, however, delivery to many tissues is limited. Efforts have been made to improve the efficiency of gene transfer into target organ
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69f2a514abba2bababf04b57921a865d