'Safe harbor' gene therapy approach may have first success.
case_report · Level V
Where this comes from
- Record sourced from PubMed, PMID 39818894.
- Also identified by DOI 10.1126/science.adv9833.
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Abstract
An obscure gene editor was used to restore a missing liver enzyme in an infant with a devastating metabolic condition.
Medical subject headings
- Gene Editing
- Genetic Therapy
- Metabolic Diseases