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Degenerative and inherited neurological disorders

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Molecular and Cell Biology of Human Gene Therapeutics

Part of the book series: Molecular and Cell Biology of Human Diseases Series ((Mol. Cell Biol. Hu. Dis.,volume 20))

Abstract

As of January 1995, a total of 95 human gene therapy/gene marking clinical protocols had been approved and/or presented worldwide; in addition many of these have already been started. Some 19% (18 protocols) are to treat inherited disorders, while 12% (eleven protocols) target CNS tumors (e.g. neuroblastomas and astrocytomas) (Summers, 1993; Anderson, 1994). None of the 95 protocols is to treat either inherited or sporadic neurological or neuromuscular disease proper. Also, of many new commercial gene therapy companies, only one is targeting gene therapy for neurological disease (Dodet, 1993; Summers, 1993). The current under-representation of human neurological gene therapy contrasts with its pre-eminent historical role, since human gene therapy was already proposed in 1972 for a metabolic disorder, the Lesch—Nyhan syndrome, which has devastating effects on brain function (Friedmann and Roblin, 1972). Nevertheless, conventional neurological therapies for both inherited and sporadic disorders are limited by factors such as drug side effects and the development of tolerance during long-term administration (Kopin, 1993), or technical shortcomings of neurosurgical procedures, for example due to tumor inaccessibility (Takamiya et al., 1993).

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  1. Pedro R. Lowenstein
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  1. Department of Biochemistry, Royal Holloway, University of London, Egham, Surrey, TW20 0EX, UK

    George Dickson

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Lowenstein, P.R. (1995). Degenerative and inherited neurological disorders. In: Dickson, G. (eds) Molecular and Cell Biology of Human Gene Therapeutics. Molecular and Cell Biology of Human Diseases Series, vol 20. Springer, Dordrecht. https://doi.org/10.1007/978-94-011-0547-7_16

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  • DOI: https://doi.org/10.1007/978-94-011-0547-7_16

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