A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression.
差异化的β-珠蛋白基因替换策略利用异源内含子来恢复生理表达
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.02.036
Wallace Kirby A, Gerstenberg Trevor L, Ennis Craig L, Perez-Bermejo Juan A, Partridge James R, Bandoro Christopher, Matern William M, Andreoletti Gaia, Krassovsky Kristina, Kabir Shaheen, Lalisan Cassandra D, Churi Aishwarya R, Chew Glen M, Corbo Lana, Vincelette Jon E, Klasson Timothy D, Silva Brian J, Strukov Yuri G, Quejarro B Joy, Hill Kaisle A, Treusch Sebastian, Grogan Jane L, Dever Daniel P, Porteus Matthew H, Wienert Beeke
