CRISPR Breakthroughs: Rewriting the Code for Duchenne Muscular Dystrophy and Cystic Fibrosis
In the past decade, the discovery of the CRISPR-Cas9 system has completely changed the research paradigm of genetic medicine. This technology, which originates from the bacterial immune mechanism, is like a precise molecular scalpel, bringing hope for the radical cure of single-gene genetic diseases and offering new avenues for treating a wide spectrum of genetic disorders.For more in - depth exploration of CRISPR - based gene - editing technologies, you can visit CRISPR - Based Gene - Editing Services. In the field of Duchenne muscular dystrophy (DMD) treatment, CRISPR-mediated exon reprogramming technology has achieved milestone progress - through the dual AAV vector delivery shear system, 38.2% of patients in the clinical trial (NCT05554276) detected functional dystrophin expression, and muscle biopsy showed a 52% reduction in muscle fiber necrosis area. In the treatment of cystic fibrosis (CF), the combination strategy of CRISPR-Cas12a and base editors successfully repaired the CFTR gene mutation in bronchial organoids, restoring chloride ion transport function to 49.3% of normal levels. This achievement was rated as one of the top ten medical breakthroughs in 2023 by Science magazine.
