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Gene therapy and the brain

H A Jinnah1, T Friedmann

  • 1Department of Neurology, Johns Hopkins University Hospital, Baltimore, MD, USA.

British Medical Bulletin
|January 1, 1995
PubMed
Summary
This summary is machine-generated.

Gene therapy shows promise for treating neurologic disorders like Lesch-Nyhan disease, Parkinsonism, Alzheimer

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Area of Science:

  • Neurology
  • Genetics
  • Molecular Biology

Background:

  • Gene therapy is an emerging treatment modality.
  • Four distinct neurologic disorders are considered: Lesch-Nyhan disease, Parkinsonism, Alzheimer's disease, and malignant brain tumors.

Purpose of the Study:

  • To explore the application of gene therapy concepts to four specific neurologic disorders.
  • To review the potential of gene transfer as a therapeutic strategy for these conditions.

Main Methods:

  • Review of existing research on gene therapy applications.
  • Analysis of in vitro systems and animal models for each disorder.

Main Results:

  • Lesch-Nyhan disease: A genetic disorder caused by a defect in the hypoxanthine-guanine phosphoribosyltransferase (HPRT) gene.

Conclusions:

  • Basic research suggests gene transfer tools offer a novel and potentially effective treatment strategy for these neurologic disorders.