日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency

临床前使用临床相关的 scAAV9/SUMF1 载体治疗多种硫酸酯酶缺乏症

Maximiliano Presa #, Rachel M Bailey #, Somdatta Ray, Lauren Bailey, Saurabh Tata, Tara Murphy, Pierre-Alexandre Piec, Harold Combs, Steven J Gray, Cathleen Lutz

Bone marrow transplantation increases sulfatase activity in somatic tissues in a multiple sulfatase deficiency mouse model

骨髓移植可增加多种硫酸酯酶缺乏症小鼠模型中体细胞组织中的硫酸酯酶活性

Maximiliano Presa, Vi Pham, Somdatta Ray, Pierre-Alexandre Piec, Jennifer Ryan, Timothy Billings, Harold Coombs, Lars Schlotawa, Troy Lund, Rebecca C Ahrens-Nicklas, Cathleen Lutz

New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency

具有次等位基因 SUMF1 变体的新型小鼠模型可模拟多种硫酸酯酶缺乏症的减弱形式

Nicolina Cristina Sorrentino, Maximiliano Presa, Sergio Attanasio, Vincenzo Cacace, Martina Sofia, Aamir Zuberi, Jennifer Ryan, Somdatta Ray, Igor Petkovic, Karthikeyan Radhakrishnan, Lars Schlotawa, Andrea Ballabio, Cathleen Lutz, Nicola Brunetti-Pierri