(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying gene cargoes because of ...

Gene therapy involves the introduction of foreign genomic materials into host cells to trigger clinical benefits. 1 Over the years, scientists have developed numerous viral and non-viral vectors to ...

Key trends include strategic CDMO partnerships and a shift to in vivo administration, enhancing accessibility and innovation ...

New research shows potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector This study describes the discovery, ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

Under the agreement, Bionova will serve as the preferred manufacturing partner for Syenex’s best-in-class delivery platforms.