Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Sophia Todeasa"'
Autor:
Michaël Hocquemiller, Laura Giersch, Xin Mei, Amanda L. Gross, Ashley N. Randle, Heather L. Gray-Edwards, Judith A. Hudson, Sophia Todeasa, Lorelei Stoica, Douglas R. Martin, Miguel Sena-Esteves, Karen Aiach, Ralph Laufer
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 281-292 (2022)
GM1 gangliosidosis is a rare, inherited neurodegenerative disorder caused by mutations in the GLB1 gene, which encodes the lysosomal hydrolase acid β-galactosidase (β-gal). β-gal deficiency leads to toxic accumulation of GM1 ganglioside, predomina
Externí odkaz:
https://doaj.org/article/157bca7427c44bc4a59c76e532caa74e
Autor:
Toloo Taghian, Ana Rita Batista, Sarah Kamper, Michael Caldwell, Laura Lilley, Hao Li, Paola Rodriguez, Katerina Mesa, Shaokuan Zheng, Robert M. King, Matthew J. Gounis, Sophia Todeasa, Anne Maguire, Douglas R. Martin, Miguel Sena-Esteves, Thomas J. Meade, Heather L. Gray-Edwards
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 128-134 (2021)
Transformative results of adeno-associated virus (AAV) gene therapy in patients with spinal muscular atrophy and Leber’s congenital amaurosis led to approval of the first two AAV products in the United States to treat these diseases. These extraord
Externí odkaz:
https://doaj.org/article/c16bf88485644845ad32d3c283989ad8