Virus as Vectors for Human Gene Therapy

Currently, gene therapy refers to the transfer of a gene that encodes a functional protein into a cell or the transfer of an entity that will alter the expression of an endogenous gene in a cell. The efficient transfer of the genetic material into a cell is necessary to achieve the desired therapeutic effect. Based on the virus life cycle, infectious virions are very efficient at transferring genetic information. Most gene therapy experiments have used viral vectors comprising elements of a virus that result in a replication-incompetent virus. In initial studies, immediate or immediate early genes were deleted. These vectors could potentially undergo recombination to produce a wild-type virus capable of multiple rounds of replication. These viral vectors replaced one or more viral genes with a promoter and coding sequence of interest. Competent replicating viral vectors were produced using packaging cells that provided deleted viral genes in trans. For these viruses, protein(s) normally present on the surface of the wild-type virus were also present in the viral vector particle. Thus, the species and the cell types infected by these viral vectors remained the same as the wild-type virus from which they were derived.

 

Reverse genetics has been used to generate mutant or reassortant influenza viruses to assess their replication, virulence, pathogenicity, host range, and transmissibility. Moreover, this technology is now being used to generate approved influenza virus vaccines and develop novel vaccines to combat seasonal and (future) pandemic influenza viruses. The generation of vaccines for highly pathogenic avian influenza viruses, including those of the H5N1 subtype, relies on reverse genetics, which allows the production of influenza viruses from cloned cDNA . The process involves the use of mammalian cells and during development of the technology, 293T cells, 293T/MDCK cell mixtures and Vero cells have been used. However, the requirement for a mammalian cell in the reverse genetics process imposes specific regulatory requirements when the rescued virus is intended for use in the development of human vaccine.

  • Vectors in human gene therapy
  • Lentiviral vectors
  • Retroviral vectors
  • Progress and problems with the use of viral vectors for gene therapy
  • Gene therapy using adeno-associated virus vectors
  • Adenoviral vectors
  • HSV genes
  • Baculoviral vectors

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