Turning genes into medicines-what have we learned from gene therapy drug development in the past decade?
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 33199678.
- Also identified by DOI 10.1038/s41467-020-19507-0 and PMC identifier 7669843.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
Gene and cell therapy products approved over the past decade in Europe and North America have provided new therapeutic options for single gene disorders and for hematologic malignancies. Lessons learned, and limitations identified, are reviewed.
Medical subject headings
- Drug Development
- Genetic Therapy