该研究也指出，由于DMD突变类型多样且分布广泛，单一ASO药物难以覆盖所有患者。尤其是针对深层内含子突变的修复，现有方法的效果仍然有限。此外，基因治疗的研发成本高昂，临床转化周期长，给患者的普及使用带来显著障碍。

Most people associate sunburns with DNA damage -- it's what we've been taught in textbooks and by dermatologists for years.

New research reveals that sunburn may be caused by RNA damage, not DNA, challenging long-held beliefs about UV exposure ...

This new tool, known as minimal, versatile genetic perturbation technology (mvGPT), combines the powers of gene editing, ...

Cai, now 46, was diagnosed with amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, back in 2019. As one of the most ...

Analysts at William Blair boosted their FY2024 earnings per share estimates for shares of Sarepta Therapeutics in a research ...

Researchers at the University of Copenhagen and NTU Singapore have discovered that RNA damage, not DNA damage, is the real trigger for the acute effec ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.

New research reveals that sunburn’s acute effects, like inflammation and cell death, are triggered by RNA damage rather than ...

Metagenomi says it hopes to start clinical trials next year to test MGX-001, its gene-editing therapy candidate for ...

Moderna has been testing an mRNA candidate for influenza viruses like the H5 and H7 strains that are seen as pandemic threats ...

Mysterious Blobs Found inside Cells Are Rewriting the Story of How Life Works Tiny specks called biomolecular condensates are ...