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Literature DB >> 30157442

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.

Ding An, Jessica L Schneller, Andrea Frassetto, Shi Liang, Xuling Zhu, Ji-Sun Park, Matt Theisen, Sue-Jean Hong, Jenny Zhou, Raj Rajendran, Becca Levy, Rebecca Howell, Gilles Besin, Vladimir Presnyak, Staci Sabnis, Kerry E Murphy-Benenato, E Sathyajith Kumarasinghe, Timothy Salerno, Cosmin Mihai, Christine M Lukacs, Randy J Chandler, Lin T Guey, Charles P Venditti, Paolo G V Martini.

Abstract

Entities: Disease

Year: 2018 PMID： 30157442 DOI： 10.1016/j.celrep.2018.08.049

Source DB: PubMed Journal: Cell Rep Impact factor: 9.423

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10 in total

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Journal: J Pers Med Date: 2021-01-28

Review 5. Chemically modified mRNA beyond COVID-19: Potential preventive and therapeutic applications for targeting chronic diseases.

Authors: Dana Elkhalifa; Menatallah Rayan; Ahmed T Negmeldin; Abdelbary Elhissi; Ashraf Khalil
Journal: Biomed Pharmacother Date: 2021-10-28 Impact factor: 6.529

6. In vivo genome editing at the albumin locus to treat methylmalonic acidemia.

Authors: Jessica L Schneller; Ciaran M Lee; Leah E Venturoni; Randy J Chandler; Ang Li; Sangho Myung; Thomas J Cradick; Ayrea E Hurley; William R Lagor; Gang Bao; Charles P Venditti
Journal: Mol Ther Methods Clin Dev Date: 2021-11-11 Impact factor: 6.698

7. Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia.

Authors: Britt Delnoy; Minela Haskovic; Jo Vanoevelen; Laura K M Steinbusch; Esther Naomi Vos; Kèvin Knoops; Luc J I Zimmermann; Marek Noga; Dirk J Lefeber; Paolo G V Martini; Ana I Coelho; Maria Estela Rubio-Gozalbo
Journal: J Inherit Metab Dis Date: 2022-05-27 Impact factor: 4.750