Review paper

Messenger RNA therapy for rare genetic metabolic diseases

Gut24.50
Volume: 68, Issue: 7, Pages: 1323 - 1330
Published: Feb 22, 2019
Abstract
Decades of intense research in molecular biology and biochemistry are fructifying in the emergence of therapeutic messenger RNAs (mRNA) as a new class of drugs. Synthetic mRNAs can be sequence optimised to improve translatability into proteins, as well as chemically modified to reduce immunogenicity and increase chemical stability using naturally occurring uridine modifications. These structural improvements, together with the development of...
Paper Details
Title
Messenger RNA therapy for rare genetic metabolic diseases
Published Date
Feb 22, 2019
Journal
Volume
68
Issue
7
Pages
1323 - 1330
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