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.
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, ...
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 ...
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 ...
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 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) ...
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 ...
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 ...
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, ...
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 ...