Gene Therapy for Human Genetic Disease?

  title={Gene Therapy for Human Genetic Disease?},
  author={Theodore Friedmann and Richard Roblin},
  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… 


  • J. Morrow
  • Biology
    Annals of the New York Academy of Sciences
  • 1976
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.

Current Status and Future Prospects of Gene Therapy

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.

Gene therapy comes of age

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.

Gene and Cell Therapy

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.

Gene therapy made difficult

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.

Gene Therapy of Neurological Disorders

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.

Gene therapy as a new treatment option for inherited monogenic diseases.

  • P. Boudes
  • Medicine, Biology
    European journal of internal medicine
  • 2014

Gene therapy: A veracity or myth!

The purpose of this review is to focus on merit and demerit of gene therapy and to provide information about its future prospective.

Progress toward human gene therapy.

Current therapies for most human genetic diseases are inadequate. In response to the need for effective treatments, modern molecular genetics is providing tools for an unprecedented new approach to



The Use of DNA for Gene Therapy—the Need, Experimental Approach, and Implications

  • H. Aposhian
  • Biology, Chemistry
    Perspectives in biology and medicine
  • 1970
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.

Dietary treatment of inborn errors of metabolism.

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.

DNA‐mediated transformation of mammalian cells in culture. Increased transforming efficiency following sonication

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.

Uptake of Protein by Mammalian Cells: An Underdeveloped Area

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.

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 enzyme

Enzyme Replacement in Fabry's Disease, an Inborn Error of Metabolism

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.

DNA and gene therapy: uncoating of polyoma pseudovirus in mouse embryo cells.

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.

Prenatal genetic diagnosis. I.