Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy-Reference-Cited by-同舟云学术

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

Published:2019-04 Issue:4 Volume:27 Page:878-889
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

DeRosa Frank,Smith Lianne,Shen Yinghua,Huang Yan,Pan Jing,Xie Hongsheng,Yahalom Barak,Heartlein Michael W.

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference67 articles.

1. Fabry disease - current treatment and new drug development;Motabar;Curr. Chem. Genomics,2010

2. Fabry disease and its cardiac involvement;Kubo;J. Gen. Fam. Med.,2017

3. Screening, diagnosis, and management of patients with Fabry disease: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference;Schiffmann;Kidney Int.,2017

4. Novel gb(3) isoforms detected in urine of fabry disease patients: a metabolomic study;Auray-Blais;Curr. Med. Chem.,2012

5. Plasma LysoGb3: A useful biomarker for the diagnosis and treatment of Fabry disease heterozygotes;Nowak;Mol. Genet. Metab.,2017

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