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Preclinical Experimental Therapeutics

Landles, C; BATES, GP; (2014) Preclinical Experimental Therapeutics. In: BATES, GP and Tabrizi, SJ and Jones, L, (eds.) Huntington's Disease. (pp. 410-461). Oxford University Press: Oxford, UK. Green open access

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Abstract

This chapter begins by reviewing the mammalian models of Huntington’s disease (HD) that have been developed using mice, rats, and a number of large animals, including sheep, pigs, and nonhuman primates. Analysis of these models, together with genetically engineered mice created through specific manipulations of the mouse genome, has provided considerable insights into the molecular pathogenesis of HD. The number of potential therapeutic targets that have been proposed for HD is considerable, and their preclinical evaluation in HD mouse models is being used to select targets that should be pursued in drug development programs. Hence, mouse models have been used extensively to validate therapeutic targets and in the preclinical testing of therapeutic strategies. The limitations of these studies are discussed, and best-practice approaches are highlighted. The chapter concludes with a summary of the gene therapy approaches that are being developed, including strategies to lower the levels of huntingtin.

Type: Book chapter
Title: Preclinical Experimental Therapeutics
ISBN: 0199929149
ISBN-13: 9780199929146
Open access status: An open access version is available from UCL Discovery
DOI: 10.1093/med/9780199929146.003.0016
Publisher version: https://doi.org/10.1093/med/9780199929146.003.0016
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Neurodegenerative Diseases
URI: https://discovery.ucl.ac.uk/id/eprint/10052891
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