Gene Therapy for Human Genetic Disease?

@article{Friedmann1972GeneTF,
  title={Gene Therapy for Human Genetic Disease?},
  author={T. Friedmann and R. Roblin},
  journal={Science},
  year={1972},
  volume={175},
  pages={949 - 955}
}
In our view, gene therapy may ameliorate some human genetic diseases in the future. For this reason, we believe that research directed at the development of techniques for gene therapy should continue. For the foreseeable future, however, we oppose any further attempts at gene therapy in human patients because (i) our understanding of such basic processes as gene regulation and genetic recombination in human cells is inadequate; (ii) our understanding of the details of the relation between the… Expand
THE PROSPECTS FOR GENE THERAPY IN HUMANS
  • J. Morrow
  • Biology, Medicine
  • Annals of the New York Academy of Sciences
  • 1976
TLDR
The proposed treatment of inherited diseases of humans by administration of genes (gene therapy) has been controversial because of its possible misuse to alter people against their will. Expand
Current Status and Future Prospects of Gene Therapy
TLDR
Allovectin-7® is a DNA plasmid containing the gene encoding human MHC class I HLA-B7, one of many examples of how gene therapy may affect cancer treatment in the near future. Expand
Gene therapy comes of age
TLDR
The pioneering work that led the gene therapy field to its current state is reviewed, gene-editing technologies that are expected to play a major role in the field's future are described, and practical challenges in getting these therapies to patients who need them are discussed. Expand
Gene and Cell Therapy
TLDR
The enormous technological advance in the field of biomedicine, with the human genome sequencing or the development of high-throughput techniques, contributed to an effective application of gene therapy in the human context. Expand
Gene therapy made difficult
TLDR
Fundamental diYculties in the basic biology of gene transfer techniques need to be addressed before real progress can be made in the application of this fashionable concept to the clinical arena. Expand
Gene therapy made difficult
TLDR
The development of a number of vector systems designed to deliver foreign genes to cells set the stage for the emergence of experimental gene transfer to investigate the function of individual genes, soon to be followed by gene therapy. Expand
Gene Therapy for Nervous System Disease a
  • M. Tuszynski
  • Medicine
  • Annals of the New York Academy of Sciences
  • 1997
TLDR
Two approaches to gene therapy have been used to date in animal models and in human clinical trials, and each approach has unique benefits and drawbacks. Expand
Gene Therapy of Neurological Disorders
TLDR
Gene therapy can now be combined with antisense techniques and RNA interference (RNAi), further increasing the therapeutic scope of gene therapy, particularly with use of genetically modified cells. Expand
Gene therapy as a new treatment option for inherited monogenic diseases.
  • P. Boudes
  • Medicine
  • European journal of internal medicine
  • 2014
TLDR
A systematic review of the literature highlights progresses and questions that remain to be answered to make gene therapy an integral part of the authors' therapeutic arsenal. Expand
Gene therapy: A veracity or myth!
TLDR
The purpose of this review is to focus on merit and demerit of gene therapy and to provide information about its future prospective. Expand
...
1
2
3
4
5
...

References

SHOWING 1-10 OF 43 REFERENCES
The Use of DNA for Gene Therapy—the Need, Experimental Approach, and Implications
  • H. Aposhian
  • Medicine
  • Perspectives in biology and medicine
  • 1970
TLDR
These basic scientific findings are the foundation for many other technical developments that are needed for the eventual treatment or cure of certain kinds of human diseases. Expand
Dietary treatment of inborn errors of metabolism.
TLDR
In this review, disorders which exemplify each of these modes of therapy are described in sufficient detail to permit an appreciation of two problems: management of the disease under discussion, and evaluation of therapy of inborn errors in general. Expand
DNA‐mediated transformation of mammalian cells in culture. Increased transforming efficiency following sonication
TLDR
A statistical analysis of 100 experiments demonstrated that the increase in the number of resistant cells after treatment with sonicated resistant DNA (R‐DNA) was statistically significant, and that the efficiency of transformation was greatly increased by sonication of the DNA. Expand
Uptake of Protein by Mammalian Cells: An Underdeveloped Area
TLDR
With labeled proteins used as models, it is found that the transport proceeds at very low rates, requires little energy, and is markedly enhanced by polybasic compounds, suggesting that macromolecules have only a limited chance to express their biological activity in target cells. Expand
Characterization of a temperature-sensitive mutant of polyoma virus.
TLDR
TS-a was found to be inhibited in its ability to produce infectious virus in mouse cells at 38.5 °; this inhibition could be overcome by infection with high multiplicites and implies that the virus must contribute some other function besides causing the “induction” of host cell DNA synthesis for neoplastic transformation to occur. Expand
Studies of the mechanism of action of the Shope rabbit papilloma virus. I. Concerning the nature of the induction of arginase in the infected cells.
These studies make plain that the Shope papilloma virus induces the production of an arginase in rabbit squamous epithelium, and provide evidence that the information for the synthesis of the enzymeExpand
Lasting changes in acriflavine-binding induced in mammalian cells by exogenous DNA.
TLDR
Cultured mammalian cells, when specifically stained for their DNA by limiting amounts of acriflavine, bound the dye in replicable quantities which were characteristic for each cell line studied, and fractional dye-binding levels remained constant over an indeterminate number of cell generations. Expand
Enzyme Replacement in Fabry's Disease, an Inborn Error of Metabolism
TLDR
Two patients with Fabry's disease were infused with normal plasma to provide active enzyme (ceramide trihexosidase) for hydrolysis of the plasma substrate, galactosylgalacto-glucosylceramide, and the amount of accumulated substrate in the plasma of these recipients decreased about 50 percent on day 10 after infusion. Expand
DNA and gene therapy: uncoating of polyoma pseudovirus in mouse embryo cells.
TLDR
Pseudovirions and polyoma virions are produced after infection of primary mouse embryo cells with polyoma virus and the implications of these experiments to the eventual use of DNA for gene therapy are discussed. Expand
Prenatal genetic diagnosis. I.
...
1
2
3
4
5
...