CRISPR gene editing has revolutionized genetics, but delivery challenges have restricted its clinical applications primarily ...

In addition, first-generation CRISPR systems (Cas9 and Cas12a) are too large for efficient in vivo delivery ... associated viral (AAV) vectors.” When targeting the PCSK9 gene in mouse liver ...

More recently, Bluebird Bio’s Zynteglo (betibeglogene autotemcel), a cell-based gene therapy ... adeno-associated virus/AAV, capsids, herpes simplex virus/HSV, and virus-like particles/VLPs ...

The same could be said for AAV gene therapy. “The old adage goes that gene therapy is all about delivery, and if you are able to get your therapy to the right tissue or cell type, they will ...

The AAV segment is ... new targets for particular gene therapy and improve molecular structures, giving researchers important information for the purpose of drug development. Overall, artificial ...

The therapy increases the amount of blood the ... Oct. 28, 2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and ...

Strategies like capsid engineering, immune modulation, and optimized gene delivery techniques have led to improved clinical outcomes and expanded the potential of AAV-based treatments. Furthermore, ...

Gene therapy involves the introduction of new genes ... α1,3-galactosyltransferase was evaluated in monkeys with CRISPR-induced primary hepatocellular carcinoma and in a phase I clinical trial.