mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblasts.

Furtado, Denzil; Cortez-Jugo, Christina; Hung, Ya Hui; Bush, Ashley I; Caruso, Frank

Furtado, Denzil; Cortez-Jugo, Christina; Hung, Ya Hui; Bush, Ashley I; Caruso, Frank.

Affiliation

Furtado D; Department of Chemical Engineering, The University of Melbourne, Parkville, Victoria 3010, Australia.
Cortez-Jugo C; Department of Chemical Engineering, The University of Melbourne, Parkville, Victoria 3010, Australia.
Hung YH; Melbourne Dementia Research Centre, The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia.
Bush AI; Melbourne Dementia Research Centre, The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia.
Caruso F; Department of Chemical Engineering, The University of Melbourne, Parkville, Victoria 3010, Australia.

Mol Pharm ; 19(11): 3987-3999, 2022 11 07.

Article in En | MEDLINE | ID: mdl-36125338

Subject(s)

Membrane Glycoproteins; Niemann-Pick Diseases; Humans; Membrane Glycoproteins/metabolism; RNA, Messenger/genetics; RNA, Messenger/metabolism; Carrier Proteins/metabolism; Intracellular Signaling Peptides and Proteins/genetics; Intracellular Signaling Peptides and Proteins/metabolism; Fibroblasts/metabolism; Cholesterol/metabolism; Niemann-Pick Diseases/metabolism; Niemann-Pick Diseases/pathology

Key words

Niemann−Pick disease type C; gene therapy; genetic disease; mRNA delivery; mRNA therapeutics; nucleic acid therapeutics

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Membrane Glycoproteins / Niemann-Pick Diseases Limits: Humans Language: En Journal: Mol Pharm Journal subject: BIOLOGIA MOLECULAR / FARMACIA / FARMACOLOGIA Year: 2022 Type: Article Affiliation country: Australia

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