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The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific ...
Scientists successfully used personalised CRISPR base editing to ease symptoms in a baby with a rare liver disorder. This ...
In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized ...
Drug development takes years, time KJ did not have. But his doctor, Rebecca Ahrens-Nicklas, a metabolic-disease expert at the ...
Despite rapid advances in genome and exome sequencing, many individuals with rare diseases remain undiagnosed. In a ...