Messenger RNA therapy for rare genetic metabolic diseases

Berraondo, P; Martini, PGV; Avila, MA; Fontanellas, A

Avila, MA; Fontanellas, A (reprint author), Univ Navarra, Hepatol Program, CIMA, Pamplona 31008, Navarra, Spain.

GUT, 2019; 68 (7): 1323

Abstract

Decades of intense research in molecular biology and biochemistry are fructifying in the emergence of therapeutic messenger RNAs (mRNA) as a new class......

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