mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts-Reference-Cited by-同舟云学术

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

Published:2022-09-20 Issue:11 Volume:19 Page:3987-3999
ISSN:1543-8384
Container-title:Molecular Pharmaceutics
language:en
Short-container-title:Mol. Pharmaceutics

Author:

Furtado Denzil¹,Cortez-Jugo Christina¹,Hung Ya Hui²,Bush Ashley I.²,Caruso Frank¹^ORCID

Affiliation:

1. Department of Chemical Engineering, The University of Melbourne, Parkville, Victoria 3010, Australia

2. Melbourne Dementia Research Centre, The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia

Funder

Friedreich's Ataxia Research Alliance

National Health and Medical Research Council

Australian NPC Disease Foundation

Friedreich Ataxia Research Association, Australia

Westpac Foundation

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Pharmaceutical Science,Molecular Medicine

Link

https://pubs.acs.org/doi/pdf/10.1021/acs.molpharmaceut.2c00463

Reference71 articles.

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4. NPC1 regulates ER contacts with endocytic organelles to mediate cholesterol egress

5. Genetic evidence for nonredundant functional cooperativity between NPC1 and NPC2 in lipid transport

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