Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
申请人:ModernaTX, Inc.
公开号:US11001861B2
公开(公告)日:2021-05-11
The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
本发明涉及用于治疗1型半乳糖血症(Gal-1)的mRNA疗法。用于本发明的mRNA在体内给药时,编码人半乳糖-1-磷酸尿苷酰转移酶(GALT)、其异构体、其功能片段以及包含GALT的融合蛋白。本发明的 mRNA 最好封装在脂质纳米颗粒(LNPs)中,以便在给药时有效地传递到受试者的细胞和/或组织中。本发明的 mRNA 疗法可增加和/或恢复受试者体内不足的 GALT 表达和/或活性水平。