The Pharmaceutical Journal
Vol 277 No 7430 p689
9 December 2006

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· TDM
· Work breaks
· Pfizer products
· Enhanced services
· Concordance (2)
· Small pharmacies
· Huntington's disease
· Section 60 Order
· The Society
· Technicians

Letters to the Editor

Huntington's disease

Gene therapy offers a promising prospect

From Dr I. I. Al-Janabi, MRPharmS

The article by Elizabeth Bevan and Carol Paton entitled “What evidence there is for the drug treatment of Huntington’s disease” (PJ, 25 November, pp641–2) highlights the problems associated with the use of conventional pharmacological agents, particularly for neurological disorders. The use of these agents in Huntington’s disease (HD) gives rise to adverse reactions and is limited to symptomatic intervention. The adverse reactions often result from these agents reaching tissues not intended to be acted on — analogous to putting out a fire in a house by flooding the whole town.

Gene therapy offers a promising prospect for the treatment of genetic diseases like HD which, within successive generations of one affected family, can appear at a younger age or a more severe form. The latter phenomenon, known as anticipation, is thought to be caused by the expansion of the CAG trinucleotide repeats of the mutant allele. Because most of the sufferers are heterozygotes for the mutant allele, one gene therapy strategy for HD is to counteract the harmful effect of the mutant protein through the degradation of its progenitor, mRNA, by the use of RNA interference (RNAi). Targeted delivery of RNAi specific for the mutant mRNA should leave the normal allele expressing the required normal protein, albeit at a reduced level.¹ This approach is currently being tested in the hope of translating the results to clinical trials.

Ismail I. Al-Janabi
Epsom, Surrey

Reference

1. Denovan-Wright EM, Davidson BL. RNAi : a potential therapy for the dominantly inherited nucleotide repeat diseases. Gene Therapy 2006;13:525–31.

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