A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR.
Zhang J, Frabutt DA, Chrzanowski M, Li N, Miller LM, Tian J, Mulcrone PL, Lam AK, Draper BE, Jarrold MF, Herzog RW, Xiao W.
Zhang J, et al.
Mol Ther Methods Clin Dev. 2024 Feb 3;32(1):101206. doi: 10.1016/j.omtm.2024.101206. eCollection 2024 Mar 14.
Mol Ther Methods Clin Dev. 2024.
PMID: 38390555
Free PMC article.
The treatment of mice with hemophilia B with cceAAV-FIX resulted in significantly enhanced long-term FIX expression. The cceAAV vectors hold several advantages over scAAV vectors, potentially leading to the development of improved human gene therapy drugs....
The treatment of mice with hemophilia B with cceAAV-FIX resulted in significantly enhanced long-term FIX expression. The cceAA …