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Patent Issued for Adeno-associated virus compositions for PAH gene transfer and methods of use thereof (USPTO 12064486).
- Source :
- Cancer Gene Therapy Week; 9/9/2024, p146-146, 1p
- Publication Year :
- 2024
-
Abstract
- The article focuses on a U.S. patent application filed by Homology Medicines Inc. for an adeno-associated virus (AAV) gene therapy aimed at treating phenylketonuria (PKU). It explains that the patent details a novel AAV composition designed to restore phenylalanine hydroxylase (PAH) gene function in PKU patients, with specific capsid proteins and regulatory elements to ensure effective gene delivery.
Details
- Language :
- English
- ISSN :
- 15436837
- Database :
- Complementary Index
- Journal :
- Cancer Gene Therapy Week
- Publication Type :
- Periodical
- Accession number :
- 179476670