In a groundbreaking first, a baby suffering from a rare genetic disorder has been treated with custom gene-editing therapy designed to fix his specific mutation. The innovative treatment involved three infusions of billions of gene-editors targeting a defect in the baby's liver, leading to a partial reversal of his condition and reducing risks like brain damage. According to Dr. Rebecca Ahrens-Nicklas, the therapy signifies transformative potential for patients lacking other options. The baby, KJ Muldoon, has shown developmental milestones since the treatment, bringing great joy to his family.
The treatment represents a groundbreaking approach in gene therapy, specifically tailored to a child's unique mutation, potentially reversing life-threatening conditions.
Dr. Ahrens-Nicklas notes the therapy's significance, stating, 'This shows the potential that we can really open the door for these transformative treatments.'
Parents of KJ Muldoon express their joy, highlighting the importance of developmental milestones: 'Seeing him reach milestones that are important for any infant... blows us away.'
#gene-editing #genetic-disorders #healthcare-innovations #medical-breakthroughs #pediatric-treatment
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