Abstract: The present disclosure provides compositions and methods for gene therapy. Further, the present disclosure provides compositions and methods for treatment of Wilson's Disease through novel gene therapy mechanisms. Wherein a composition of a closed circular cDNA integrating gene therapy construct, the gene therapy construct comprising, from 5? to 3?, a polynucleotide sequence is disclosed.
Type:
Application
Filed:
October 18, 2022
Publication date:
May 8, 2025
Applicant:
LogicBio Therapeutics, Inc.
Inventors:
Shengwen Zhang, Jing Liao, Shaobin Wang