Viral Vectors
The advancement of precision medicine, including gene therapy and gene-editing technologies, has shown promising potential in the treatment of genetic disorders. One such tool is the viral vector, which delivers transgenes into target cells or tissues. Viral vectors are commonly used to deliver genetic materials to cells in vivo or in vitro. These tools rely on viruses that have specialized molecular mechanisms to transport their genomes to the cells they infect. In addition, some properties that need to be taken into account when using a virus as a viral vector include safety, low toxicity, stability, and sometimes cell specificity. Some current viral vectors that have been used in academic research, gene therapies, and/or vaccines include, adeno-associated viruses, lenti/retroviruses, the baculoviruses, and most recently, the Herpes Simplex virus
Gene Delivery
Gene delivery techniques serve as the important groundwork for a wide range of research and clinical applications, including gene therapy. Introducing foreign genetic materials into cells allows us to study functions of the code of life and to develop many sophisticated tools, such as the CRISPR-Cas system and optogenetic tools, for further research and real-world applications in medical, environmental, and agricultural.
Gene therapies
Gene therapy is a technique that modifies a persons to treat or cure genetic disorders, such as Sickle Cell anemia or Hemophilia, by repairing or reconstructing damaged genetic material. This type of therapeutic requires a gene delivery vehicle such as a viral vector.Viral vectors are commonly used for this application since we are naturally host to some viruses that are used. However, some challenges included the vector causing a overly immunogenic reaction which would make the vector no longer effective for treatment or cytotoxic to the cells resulting in cell death. Non-viral vectors have also been developed for this application. In addition, there are currently no approved gene therapy treatments; however there are many in clinical trails.
Comparison of Common Viral Vectors to Herpes Simplex Virus
Adeno-associated Viruses
ssDNA genome
High titer and transduction efficacy
Limited transgene capacity (~5 kb)
Possible integration into the host genome
Lentiviruses
ssDNA genome
Wide host-cell range
High Transduction efficacy
Limited transgene capacity (~8kb)
Possible Integration into the host genome
Herpes Simplex Virus
dsDNA genome of 150 kb
>30 kb transgene capacity
Naturally infects human nervous system
No integration into the host genome
Main safety concern: cytotoxicity
Leader: Wasu Ngamkanjanarat