杜氏肌营养不良症（Duchenne muscular dystrophy，DMD）是由编码肌营养不良蛋白（dystrophin，也被成为抗肌萎缩蛋白）的基因DMD（该基因长约220万bp ...

The Federal Reserve rate cuts have sent the market up, but some stocks are rising more than others. That includes small ...

通过AAV载体对aTdCBE进行体内递送，对DMD外显子55剪接位点进行编辑，成功地实现了DMD外显子55跳跃，并恢复了小鼠肌肉组织中dystrophin蛋白的表达，为杜氏肌营养不良（DMD）的治疗带来了新技术。 目前蛋白定向进化方法主要包括噬菌体辅助的持续演化技术（PACE ...

A groundbreaking study led by researchers from the University of Ottawa’s Faculty of Medicine offers promising insights that ...

日前，昆明理工大学灵长类转化医学研究院、省部共建非人灵长类国家重点实验室、昆明理工大学生命科学与技术学院陈永昌、季维智团队，与广州实验室、上海交通大学医学院附属新华医院等机构合作，应用非人灵长类疾病模型解析杜氏肌营养不良症（Duchenne ...

Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, the ...

While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, ...

Sarepta's Elevidys approval, revenue growth projections, and competitive position make it a strong investment in DMD ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...

Dyne (DYN) stock fell 31% after the company announced data for its drug DYNE-251 in the treatment of Duchenne muscular ...

Endpoints can include changes in levels of the therapeutic protein produced by the delivered gene (for example, Factor VIII or Factor IX for haemophilia A and B, respectively, or mini-dystrophin ...