Skeletal muscle-directed gene therapy: hijacking the fusogenic properties of muscle cells  

作　　者：Hildegard Buning Michael Morgan Axel Schambach 

机构地区：[1]Institute of Experimental Hematology,Hannover Medical School,Hannover,30625,Germany [2]REBIRTH Research Center for Translational Regenerative Medicine,Hannover Medical School,Hannover,30625,Germany [3]Division of Hematology/Oncology,Boston Children’s Hospital,Harvard Medical School,Boston,MA,02115,USA

出　　处：《Signal Transduction and Targeted Therapy》2023年第10期4428-4430,共3页信号转导与靶向治疗（英文）

摘　　要：A recent study published in Cell by the Millay lab reports on an elegant strategy to expand the repertoire of lentiviral(LV)vectors from being the main delivery tool for ex vivo gene therapy to an in vivo applicable viral vector that specifically transduces skeletal muscle.1 This very significant advance may result in novel therapeutics for patients suffering from various skeletal muscle diseases.

关 键 词：SKELETAL directed MUSCLE 

分 类 号：R746[医药卫生—神经病学与精神病学]