AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

Michaël Hocquemiller,Kim M. Hemsley, Meghan L. Douglass,Sarah J. Tamang,Daniel Neumann,Barbara M. King,Helen Beard,Paul J. Trim,Leanne K. Winner,Adeline A. Lau,Marten F. Snel,Cathy Gomila,Jérôme Ausseil,Xin Mei,Laura Giersch,Mark Plavsic,Ralph Laufer

Molecular Therapy - Methods & Clinical Development（2020）

引用 20|浏览17

暂无评分

关键词

AAV,mucopolysaccharidosis,gene therapy,lysosomal storage disease

AI 理解论文

溯源树

样例

生成溯源树，研究论文发展脉络

Chat Paper

正在生成论文摘要