This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

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 ...

Gene therapy is defined as the treatment of disease by transfer of genetic material into cells, to prevent, treat and potentially even cure a disease. Gene therapies can work by several mechanisms: 1) ...

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, ...

Researchers develop a nanosensing platform that can assess the quality of individual viral vector particles Viral vectors hold much potential for gene editing and gene therapy, but there is a pressing ...

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 ...

Viral vectors are critical vehicles for the delivery of life-saving cell and gene therapies. Adeno-associated virus (AAV) and lentiviral vectors (LVV), two of the most widely used vector platforms, ...

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 ...

Pointing to a few empty chairs in a conference room, moderator David T. Curiel joked that a symposium on emerging nonviral vectors was a "failure" compared to other packed talks at the American ...

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 ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Viral vectors are engineered viruses that are used to deliver genetic material into cells for various applications, such as gene therapy, vaccine development, and biotechnology research. These viruses ...