Gene and cell therapy is moving fast – and in a clear direction. Programs are pushing toward more complex payloads, virus-free engineering, and manufacturing models that can scale from early research ...

The genetic medicine revolution is underway. Just 18 months ago, very few people in the general population had heard of mRNA vaccines; now, hundreds of millions globally have been vaccinated with one.

The researchers say that, to their knowledge, this is the first demonstration of programmable, site-specific integration of a ...

Molecular cloning relies on two key components to construct recombinant DNA: 1) a DNA vector and 2) one or more DNA fragments containing the gene(s) of interest (GOI). To produce a GOI fragment, DNA ...

What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...

New research shows epigenetic silencing may limit durability of AAV gene therapy in haemophilia by reducing long term transgene expression in liver cells.