A short course on virology / vectorology / gene therapy.

  title={A short course on virology / vectorology / gene therapy.},
  author={Z. Debyser},
  journal={Current gene therapy},
  volume={3 6},
  • Z. Debyser
  • Published 2003
  • Biology, Medicine
  • Current gene therapy
For people starting off in the field of gene therapy, the encountered terminology is often quite confusing. Moreover, the background on basic virology may be modest. The following introduction provides a head start to any novice willing to gain more in-depth knowledge on the subject. The development of gene therapy is also addressed from a historical perspective. 
Progresses towards safe and efficient gene therapy vectors
This paper reviews the major gene delivery vectors and recent improvements made in their design meant to overcome the issues that commonly arise with the use of gene therapy vectors. Expand
Production of Lentivirus for the Establishment of CAR-T Cells.
A typical protocol for the production of lentivirus, concentration by ultracentrifugation and determination of virus titer is presented, which can then be used in laboratory assays of gene transfer, including the establishment of CAR T cells. Expand
Controlled Genetic Manipulations
Different approaches to regulation of drug-regulatable site-specifi c recombinases rely on the fusion of the recombinase with the ligand-binding domain (LBD) of a steroid receptor, with the resulting Cre-LBD molecule regulated by the cognate steroid. Expand
In vivo manipulation of gene expression in non-human primates using lentiviral vectors as delivery vehicles.
The lentiviral vectors that are used to both express transgenes and suppress expression of endogenous genes via RNA interference (RNAi) in NHPs are described and the safety features of currently available vectors are discussed, especially important when lentiviruses are used in a species as closely related to humans as NHPs. Expand
Fetal gene therapy: Balancing ethical theory, scientific progress and the rights of others
This thesis examines the relationship between rights and duties in the field of fetal gene therapy and assesses if the current regulatory position within England and Wales is compatible with theExpand
Microfluidic approaches for gene delivery and gene therapy.
The development and application of microfluidic systems are producing increased efficiency in gene delivery and promise improved gene therapy results. Expand
The role of bone morphogenetic protein 7 in cortical neurogenesis
The role of BMP7 in cortical neurogenesis was examined during embryonic development in mice and neuronal differentiation was enhanced in cortical cultures following treatment with low concentration recombinant human B MP7, whereas higher concentrations enhanced glial differentiation. Expand
Monoliths in Bioprocessing


Gene therapy: trials and tribulations
A key problem in gene therapy is the lack of a vector system that fulfils all the requirements for safety and efficacy, and viral vectors are the most promising vectors at this time. Expand
The basic science of gene therapy.
A large number of key technical issues need to be resolved before gene therapy can be safely and effectively applied in the clinic, and future technological developments will be critical for the successful practice of gene therapy. Expand
Viral vectors in gene therapy.
  • A. Smith
  • Biology, Medicine
  • Annual review of microbiology
  • 1995
The challenge for the therapeutic use of viral vectors is to achieve efficient and often extended expression of the exogenous gene while evading the host defenses. Expand
US gene therapy in crisis.
The recent death of a young man, Jesse Gelsinger, in a gene therapy experiment has sent shock waves throughout the US research community. This tragic event has raised new questions about theExpand
Adenovirus and Adeno‐Associated Virus as Vectors for Gene Therapy a
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.
A gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells, which provided full correction of disease phenotype and clinical benefit. Expand
Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction.
These studies demonstrate the feasibility and safety of using retroviral gene transduction for human gene therapy and have implications for the design of TIL with improved antitumor potency, as well as for the possible use of lymphocytes for the gene therapy of other diseases. Expand
Adaptation of a retrovirus as a eucaryotic vector transmitting the herpes simplex virus thymidine kinase gene.
Great general applicability of retroviruses as eucaryotic vectors is suggested, as the chimeric MLV-tk virus showed single-hit kinetics in infections and behaved like known replication-defective retrovirus. Expand
1 The Origins, Evolution, and Directions of Human Gene Therapy
It is shown that gene therapy has become a dominant concept in the treatment of disease long before it has given its first compelling evidence of true clinical effectiveness, and is an established, powerful, and inexorable driving force in modern medicine. Expand
The origins, evolution and directions of human gene therapy retroviral vectors lentiviral vectors structure and genetic organization of adenovirus vectors adenovirus vectors strategies to adaptExpand