Polynucleotides encoding Citrin for the treatment of Citrullinemia type 2
Paolo Martini, Boston, MA (US); Stephen Hoge, Cambridge, MA (US); Kerry Benenato, Cambridge, MA (US); Vladimir Presnyak, Manchester, NH (US); Iain McFadyen, Medford, MA (US); Ellalahewage Sathyajith Kumarasinghe, Cambridge, MA (US); Jingsong Cao, Cambridge, MA (US); Lin Tung Guey, Lexington, MA (US); and Staci Sabnis, Cambridge, MA (US)
Assigned to ModernaTX, Inc., Cambridge, MA (US)
Filed by ModernaTX, Inc., Cambridge, MA (US)
Filed on Jan. 21, 2021, as Appl. No. 17/154,325.
Application 17/154,325 is a continuation of application No. 16/302,360, granted, now 10,993,918, previously published as PCT/US2017/033421, filed on May 18, 2017.
Claims priority of provisional application 62/338,479, filed on May 18, 2016.
Prior Publication US 2022/0071915 A1, Mar. 10, 2022
1. An mRNA comprising an open reading frame (ORF) encoding the human Citrin polypeptide of SEO ID NO: 1, wherein the ORF is 100% identical to the nucleotide sequence of SEQ ID NO:129.