北京时间2024年12月11日，西湖大学、西湖实验室吴建平团队联合闫浈团队在Nature在线发表题为“Structure and assembly of the dystrophin glycoprotein ...

最初出现轻微运动障碍，跑步、跳跃等动作变得吃力，后期逐渐出现无法行走、肢体无法正常抬起，需要借助轮椅行动，最终可能因呼吸衰竭或心力衰竭而死亡……这段描述描写的是，杜氏肌营养不良症患者的一生。杜氏肌营养不良症（Duchenne Muscular ...

Duchenne muscular dystrophy (DMD) is one of the most common and devastating childhood genetic disorders, affecting ...

从微观领域，开发基因治疗方案也有了新线索。目前针对DMD患者的治疗手段非常有限，一种通用策略是通过基因治疗来修补功能异常的肌营养不良蛋白。但是，这个蛋白的基因是人体最长的基因之一，完整递送如此长的基因去替换致病基因十分困难。

EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.

Dystrophin and β-dystroglycan are components of the dystrophin–glycoprotein complex (DGC), a multimolecular assembly that spans the cell membrane and links the actin cytoskeleton to the ...

Avidity Biosciences' AOC platform shows promising results for genetic diseases, making it a strong buy with potential upside ...

Children born with the degenerative disease have a genetic mutation that stops them producing dystrophin, a protein that is vital for muscle strength and function. The same disorder also occurs in ...

Sarepta's exon-skipping treatments, EXONDYS 51, VYONDYS 53, and AMONDYS 45, are designed to skip specific exons in the dystrophin gene, enabling the production of a functional, albeit shortened, ...

These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...

The one-dose gene therapy – much like rivals from Pfizer and Solid Biosciences – codes for a shortened form of dystrophin that is deficient in patients with the X-linked muscle-wasting ...

US biotech Sarepta will continue to be responsible for clinical development and manufacturing of the drug SRP-9001, also known as (AAVrh74.MHCK7.micro-dystrophin),with global clinical development ...