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 ...

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 ...

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 ...

Accurate characterization of large viral vectors is a critical step in developing viral vector-based gene therapies and vaccines for use in cancer immunotherapies, genetic disorders, infectious ...

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 ...

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 ...

There is perhaps no better example of getting to the root of a problem than gene therapy. This medical treatment alters the very building blocks of life, and it has the potential to treat or cure not ...