Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy

Fabry disease is a lysosomal storage disorder caused by the deficiency of α-galactosidase A. Enzyme deficiency results in a progressive decline in renal and cardiac function, leading to cardiomyopathy and end-stage renal disease. Current treatments available, including enzyme replacement therapies,...

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Detalles Bibliográficos
Autores principales: DeRosa, Frank, Smith, Lianne, Shen, Yinghua, Huang, Yan, Pan, Jing, Xie, Hongsheng, Yahalom, Barak, Heartlein, Michael W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6453518/
https://www.ncbi.nlm.nih.gov/pubmed/30879951
http://dx.doi.org/10.1016/j.ymthe.2019.03.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6453518/
https://www.ncbi.nlm.nih.gov/pubmed/30879951
http://dx.doi.org/10.1016/j.ymthe.2019.03.001

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