Baby's Life Saved by CRISPR Gene Therapy: A Medical Miracle

Baby's Life Saved by CRISPR Gene Therapy: A Medical Breakthrough A groundbreaking medical success story unfolded at Children's Hospital of Philadelphia, where baby KJ Muldoon was successfully treated for CPS1 deficiency, a rare genetic disorder often fatal for half of affected infants. Doctors employed custom CRISPR gene therapy, targeting the exact mutation in KJ's DNA. "We fought really hard to get him here. And now that he is here, I want to give him the opportunity to fight for himself," shared KJ's mother, Nicole, in an emotional interview with CBS News. The experimental therapy, developed and administered by the medical team, involved three infusions to KJ's liver. Doctors Rebecca Ahrens-Nicklas and Kiran Musunuru played key roles in this medical marvel. The success story offers hope for families facing similar challenges, showcasing the potential of advanced gene editing technology. The treatment's success is remarkable, considering the complexity of the human genome. Dr. Musunuru explained, "In six months, we were able to actually make the drug, the gene-editing therapy specifically for this patient." The procedure focused on correcting a single defective gene among 20,000 genes and 3 billion letters of DNA. While doctors continue to closely monitor KJ, so far, there have been no serious side effects. The family looks forward to celebrating KJ's first birthday in August and bringing him home soon. This medical breakthrough underscores the potential of CRISPR technology in treating genetic diseases, offering a beacon of hope for countless families.