Novel Treatment Strategy for Patients With Urea Cycle Disorders: Pharmacological Chaperones Enhance Enzyme Stability and Activity in Patient-Derived Liver Disease Models
尿素循环障碍患者的新型治疗策略:药理学伴侣增强患者来源肝病模型中酶的稳定性和活性
期刊:Journal of Inherited Metabolic Disease
影响因子:3.8
doi:10.1002/jimd.70043
Ramosaj, Adhuresa; Borsuk, Mariia; Underhaug, Jarl; Mathis, Déborah; Matsumoto, Shirou; Keogh, Adrian; Banz, Vanessa; Pandey, Amit V; Gougeard, Nadine; Rubio, Vicente; Martinez, Aurora; Allegri, Gabriella; Poms, Martin; Thöny, Beat; Häberle, Johannes; Laemmle, Alexander