In a groundbreaking development, a baby has received a personalized CRISPR gene-editing treatment to combat a severe genetic disease. KJ Muldoon, a ten-month-old baby born with a rare condition that hindered his ability to metabolize proteins, has become the first individual to undergo a tailored CRISPR therapy. This innovative treatment was carried out at the Children’s Hospital of Philadelphia.
The unique gene-editing therapy was specifically designed to correct the mutation in Muldoon’s genetic sequence that was responsible for his metabolic disorder. Although the treatment has shown promising results so far, it is still too early to determine if it has completely cured the baby’s condition. Dr. Rebecca Ahrens-Nicklas, one of Muldoon’s physicians, emphasized the importance of continued observation and research to fully understand the long-term effects of the therapy.
A team of international clinicians and researchers collaborated to develop and administer Muldoon’s personalized CRISPR treatment within a remarkably short period of six months. The therapy, detailed in a recent publication, is tailored to Muldoon’s specific genetic makeup and is unlikely to be used for any other individual in the future. Despite the challenges and uncertainties surrounding the widespread application of personalized gene-editing therapies, this groundbreaking achievement has set a precedent for treating ultra-rare genetic diseases using innovative technologies.
