Therapeutic AASS inhibition by AAV-miRNA rescues glutaric aciduria type I severe phenotype in mice
利用AAV-miRNA进行AASS治疗性抑制可挽救小鼠戊二酸尿症I型严重表型。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.07.022
Segur-Bailach, Eulàlia; Mateu-Bosch, Anna; Bofill-De Ros, Xavier; Parés, Marta; da Silva Buttkus, Patricia; Rathkolb, Birgit; Gailus-Durner, Valérie; Hrabě de Angelis, Martin; Moeini, Pedram; Gonzalez-Aseguinolaza, Gloria; Tort, Frederic; Ribes, Antonia; van Karnebeek, Clara D M; García-Villoria, Judit; Fillat, Cristina
