Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia-Reference-Cited by-同舟云学术

Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia

Published:2019-07 Issue: Volume:45 Page:519-528
ISSN:2352-3964
Container-title:EBioMedicine
language:en
Short-container-title:EBioMedicine

Author:

An Ding,Frassetto Andrea,Jacquinet Eric,Eybye Marianne,Milano Joseph,DeAntonis Christine,Nguyen Vi,Laureano Rodrigo,Milton Jaclyn,Sabnis Staci,Lukacs Christine M.,Guey Lin T.

Funder

Moderna, Inc.

Publisher

Elsevier BV

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Reference50 articles.

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4. Methylmalonic and propionic acidemias: clinical management update;Fraser;Curr Opin Pediatr,2016

5. Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia;Baumgartner;Orphanet J Rare Dis,2014

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