Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope

Children’s Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the use of personalized antisense oligonucleotides (ASOs). This innovative approach has shown promising results in preclinical evaluations, which offers new hope for patients with previously untreatable conditions and validates personalized therapies for patients in only eight weeks, significantly faster and more cost-effective than the industry average.