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, ...

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 ...

With this information in hand, researchers who are developing a gene therapy to treat a muscle condition, for instance, could use the atlas to identify AAV vectors that preferentially target the ...

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 ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Ring Therapeutics’ hunt for new human viruses that can deliver gene therapies multiple times in a single patient appears to have landed on a viable candidate, new mouse data have suggested. In a ...

To generate successful gene therapies that are capable of delivering nucleic acids to specific cells and tissues, scientists must carefully design their products. They can employ either recombinant ...

"Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline 2025"Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies are Nanoscope Therapeutics, REGENXBIO, Astellas Gene Therapy, ...

Gene therapies are unlocking new drug targets that were previously thought undruggable. Covering modalities like ASOs, siRNA, saRNA, mRNA, and ADARs, each with different mechanisms of action, gene ...

Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes ...

Infected with Wolbachia, the insects are expected to reduce the spread of dengue and Zika. But scientists say the approach may have limitations. A team of scientists confirm Wolbachia can prevent ...