Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis.
验证高灵敏度检测方法在GM1神经节苷脂病患者脑脊液和血清β-半乳糖苷酶活性定量中的应用
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2024.101318
Quadrini Karen J, Vrentas Catherine, Duke Christian, Wilson Chris, Hinderer Christian J, Weinstein David A, Al-Zaidy Samiah A, Browne Susan E, Wilson James M, Ni Yan G