申请人:AMICUS THERAPEUTICS INC
公开号:WO2021072372A1
公开(公告)日:2021-04-15
Provided herein are novel IGF2 peptides, fusion proteins, and nucleic acid sequences encoding novel IGF2 peptides and fusion proteins for the treatment of lysorsomal storage disorders, wherein the IGF2 peptides confer enhanced properties, such as enhanced expression, secretion and cellular uptake. The constructs provided herein are useful in treating lysosomal storage disorders by both enzyme replacement therapy and gene therapy.