Researchers have reported the first use of CRISPR technology in a personalized therapy for a patient with a rare genetic liver disease. A team from the Children's Hospital of Philadelphia and the University of Pennsylvania developed a treatment for KJ, who struggles to process proteins due to genetic mutations causing dangerous ammonia buildup. Unlike previous CRISPR approvals, KJ's therapy is bespoke and tailored specifically to him. However, the team hopes to develop a more universal method that could address other genetic disorders in the future.
The scientists developed a CRISPR treatment for a boy named KJ, who was born with genetic mutations in his liver cells that prevent him from breaking down proteins properly.
This drug was designed and made for KJ, so in reality this drug will probably never be used again, says Ahrens-Nicklas of the bespoke nature of the therapy.
While the therapy was created for him, the team is hopeful that the process can be made more universal and applied to other genetic mutations.
Researchers report on the first use of the gene-editing technology CRISPR in a customized therapy designed to treat one patient with a rare disease.
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