Enzyme replacement therapy for CLN1 batten disease that crosses the blood-brain-barrier
用于治疗可穿过血脑屏障的CLN1巴顿病的酶替代疗法
期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2026.109733
Raman, Renuka; Horst, Ben; Shahrokh, Zahra; Hatambeygi, Nader; Zare, Maryam; Leszczyniecka, Magdalena; Harris, Joshua S; Banks, William A; Hansen, Kim M; Erickson, Michelle A; Ekins, Sean