Gene Therapy Death Prompts Review of Adenovirus Vector

  title={Gene Therapy Death Prompts Review of Adenovirus Vector},
  author={Eliot Marshall},
  pages={2244 - 2245}
For the past 3 months, faculty and staff members at the University of Pennsylvania9s Institute for Human Gene Therapy have been trying to understand why a relatively fit 18-year-old with an inherited enzyme deficiency died on 17 September, 4 days after doctors at Penn injected a genetically altered virus into his liver. Last week, they presented their data to a government advisory committee. The patient was the first in a gene therapy trial to die of the therapy itself; his death is the latest… Expand
Approaches to improving the kinetics of adenovirus-delivered genes and gene products.
Adenovirus (Ad) vectors have been expected to play a great role in gene therapy because of their extremely high transduction efficiency and wide tropism, but due to the intrinsic deficiency of their immunogenic toxicities, their potential for gene therapy application is limited. Expand
Progress and problems with the use of viral vectors for gene therapy
With the development of a leukaemia-like syndrome in two patients cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go. Expand
Improvements in adenoviral vector technology: overcoming barriers for gene therapy
  • R. Parks
  • Biology, Medicine
  • Clinical genetics
  • 2000
The following review describes the development and characterization of a relatively new class of Ad-based vectors termed ‘gutted’ or helper-dependent adenovirus vectors (hdAds), which are deleted of all Ad protein coding sequences which, in itself, has eliminated most of the deleterious characteristics associated with Ad vectors. Expand
Gene therapy of monogenic and cardiovascular disorders
The experience from a number of clinical trials has taught us that gene transfer is technically feasible, but that the gene delivery vehicles for the transfer of genetic material are still suboptimal and that treatment may have severe side effects. Expand
Somatic Gene Therapy Using Viral Vectors: Theoretical and Clinical Implications in Relation to Treatment of Genetic Conditions in Humans
The presented studies demonstrate that the gene therapy community has succeeded in turning infectious agents into vehicles of therapeutics for treatment or amelioration of the disease phenotype, giving significant reassurance that gene therapy will become standard care for a number of individual disorders. Expand
Virus-mediated gene delivery for human gene therapy.
  • M. Giacca, S. Zacchigna
  • Biology, Medicine
  • Journal of controlled release : official journal of the Controlled Release Society
  • 2012
The current status of viral gene transfer for clinical applications is summarized, with special emphasis on the molecular properties of the major classes of viral vectors and the information so far obtained from gene therapy clinical trials. Expand
Gene therapy to the kidney using viral vectors.
The challenges and important developments regarding gene therapy to the kidney are discussed, and the recent successes and failures are related to the future potential of gene therapy as a treatment modality in the context of pediatric disease. Expand
[Gene therapy: current status and promise].
  • Y. Kaneda
  • Medicine
  • Nihon yakurigaku zasshi. Folia pharmacologica Japonica
  • 2001
It is necessary to reevaluate what barriers in tissues affect in vivo gene transfection and how to solve these problems for gene therapy, and in Japan, there should be more extensive preparation of social systems to promote clinical trials based on basic research. Expand
Gene therapy with viral vectors.
The results of these studies form the basis for the first clinical trials for correcting genetic disorders using retroviral, adenoviral, and adeno-associated viral vectors. Expand
New Directions in Pulmonary Gene Therapy.
Current developments in gene therapy for cystic fibrosis, alpha-1 antitrypsin deficiency, and surfactant protein deficiencies are summarized and the challenges of delivery to the lung are reviewed. Expand