Paolo MARTINI,Stephen G. HOGE,Kerry BENENATO,Vladimir PRESNYAK,Iain MCFADYEN,Ellalahewage Sathyajith KUMARASINGHE,Xuling ZHU,Lin Tung GUEY,Staci SABNIS
申请号：
US16570351
公开号：
US20200149052A1
申请日:
2019.09.13
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
