CRISPR-Cas9 was implemented for the first time in June of 2025 in a personalized gene editing treatment for a baby with CPS1 (carbamoyl phosphate synthetase 1) deficiency. KJ was given two doses of a specialized therapy at 7 and 8 months old, showing improvements within two months. With CRISPR gene-editing technology, a team at the University of Pennsylvania formulated a treatment using LNPs (lipid nanoparticles) to deliver a base editor capable of correcting KJ’s unique genetic mutation. The success of KJ’s case highlights the potential of gene editing therapies to transform medicine and manufacture solutions for rare diseases.
Specialized treatments with CRISPR provide a novel solution treating debilitating genetic mutations, yet the insurmountable price tag makes it unattainable for the vast majority. The highly powerful tool is still new, with the long term effects remaining unknown. However, CRISPR-based solutions provide the opportunity to address previously untreatable diseases. With the selective ability of CRISPR-Cas 9, it is the hope that such personalized treatments will become the standard of care in the future.
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https://oncodaily.com/blog/crispr-297474
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