Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

The re-transcribed DNA is then integrated into the genome of the host cell (Figure 2). This process requires the absence of the nuclear membrane and thus is restricted to the M-phase of proliferating ...

Cell culture media specialized for gene therapy production are uniquely designed to support the growth, viability, and productivity of cells used to manufacture gene therapy products, such as viral ...

Amid a broader push to beef up cell and gene therapy manufacturing capacity in Europe, Novartis has opened the doors to a €40 million ($41.2 million) viral vector facility in Mengeš, Slovenia. The new ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...