Hemophilia as a blueprint for gene therapy.
Where this comes from
- Record sourced from PubMed, PMID 34591611.
- Also identified by DOI 10.1126/science.abg0856.
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Abstract
Advances in hemophilia gene therapy could enable progress in other inherited disorders.
Medical subject headings
- Factor IX
- Factor VIII
- Genetic Therapy
- Hemophilia A