Progress in human gene therapy

Frederick J. Feuerbach, Ronald Crystal

Research output: Contribution to journalArticle

9 Citations (Scopus)

Abstract

There have been major advances in our understanding of the molecular biology and pathophysiology of human disease over the past decade. This expanding knowledge of the basic mechanisms of human disease coupled with the development of efficient methods of transferring genes to mammalian cells has stimulated considerable interest in treating diseases with gene therapy. Clinical trials have demonstrated that human gene transfer is possible, that several strategies exist for successfully introducing exogenous genes to human cells, that a variety of transgenes can evoke biologic responses important to human diseases, and that gene transfer can provide valuable insights into the pathophysiology of human disorders. Initial clinical trials have yielded encouraging results and adverse reactions have been uncommon. Despite the advances in human gene transfer, significant obstacles need to be overcome before it becomes a firmly established therapeutic option for human illness. The potential benefits of utilizing human gene therapy in the treatment of hereditary and acquired disease have generated interest in further advancing this remarkable technology.

Original languageEnglish
Pages (from-to)1791-1794
Number of pages4
JournalKidney International
Volume49
Issue number6
DOIs
Publication statusPublished - 1 Jan 1996
Externally publishedYes

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Genetic Therapy
Genes
Clinical Trials
Inborn Genetic Diseases
Transgenes
Molecular Biology
Technology
Therapeutics

ASJC Scopus subject areas

  • Nephrology

Cite this

Progress in human gene therapy. / Feuerbach, Frederick J.; Crystal, Ronald.

In: Kidney International, Vol. 49, No. 6, 01.01.1996, p. 1791-1794.

Research output: Contribution to journalArticle

Feuerbach, Frederick J. ; Crystal, Ronald. / Progress in human gene therapy. In: Kidney International. 1996 ; Vol. 49, No. 6. pp. 1791-1794.
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