Phase 1/2 Trial of Brogidirsen: Dual-Targeting Antisense Oligonucleotides for Exon 44 Skipping in Duchenne Muscular Dystrophy

Medicine, Medical Laboratory Science, Neurology, Life Sciences, Biochemistry, Bioengineering, Immunology, Genetics,
Duchenne muscular dystrophy   exon skipping   antisense oligonucleotide   dual-targeting   Brogidirsen   biomarkers   muscle function stabilization  

New Advances in Dual-Targeting Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy: Phase 1⁄2 Clinical Trial of Brogidirsen Background Duchenne Muscular Dystrophy (DMD) is a fatal genetic disorder primarily affecting skeletal and cardiac muscles, leading to early loss of mobility and, eventually, organ failure. Currently, there is no ...

Rapid and Scalable Personalized ASO Screening in Patient-Derived Organoids

Medicine, Life Sciences, Cell Biology, Biochemistry, Bioengineering, Genetics,
personalized therapeutics   antisense oligonucleotides   rare diseases   organoid models   gene therapy   Duchenne muscular dystrophy   stem cells  

Establishment of a Personalized Antisense Oligonucleotide (ASO) Screening Platform Based on Patient-Derived Organoids Academic Background In recent years, with the rapid development of genome sequencing technologies, an increasing number of rare genetic diseases have been found to be associated with specific gene mutations. Antisense oligonucleotid...