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THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.

Transactions of the American Clinical and Climatological Association - 1 Jan 2023

High Katherine A

Abstract excerpt

The journey from in vitro transfer of genes into mammalian cells to approved gene therapy products has spanned decades. This manuscript summarizes hurdles encountered and obstacles overcome in the development of successful adeno-associated viral (AAV) vectors for hemophilia B and for an inherited retinal dystrophy caused by mutations in the RPE65 gene. In the case of hemophilia B, careful analysis of the first...

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PMID: 37701622
PMCID: PMC10493758

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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