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Randomized Controlled Trial
. 2008 Apr;145(4):483-6.
doi: 10.1007/s10517-008-0124-4.

Gene therapy of amyotrophic lateral sclerosis

Affiliations
Randomized Controlled Trial

Gene therapy of amyotrophic lateral sclerosis

I A Zavalishin et al. Bull Exp Biol Med. 2008 Apr.

Abstract

Two-year experiments were performed to evaluate the neurotrophic effect of hypoxia-inducible factors (vascular endothelial growth factor and angiogenin) expressed in recombinant human adenoviruses in amyotrophic lateral sclerosis. Randomized placebo-controlled trial demonstrated safety and good tolerability of the recombinant antiviral drugs. The life span of patients under conditions of hypoxia increased after treatment with the test drug, which was probably related to improved resistance of motoneurons. The presence of virus-neutralizing antibodies decreases the effectiveness of adenoviral vectors, which necessitates differential approach to the selection of patients and continuous monitoring of gene therapy.

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