Gene therapy of amyotrophic lateral sclerosis

• Published in: Bulletin of experimental biology and medicine Vol. 145; no. 4; pp. 483 - 486
• Main Authors: Zavalishin, I. A, Bochkov, N. P, Suslina, Z. A, Zakharova, M. N, Tarantul, V. Z, Naroditskiy, B. S, Suponeva, N. A, Illarioshkin, S. N, Shmarov, M. M, Logunov, D. Y, Tutyhina, I. L, Verkhovskaya, L. V, Sedova, E. S, Vasiliev, A. V, Brylev, L. V, Ginzburg, A. L
• Format: Journal Article
• Language: English
• Published: Boston Boston : Springer US 01.04.2008; Springer US; Springer Nature B.V
• Subjects: adenoviral vector; Adenoviridae - genetics; Adult; amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - genetics; Amyotrophic Lateral Sclerosis - mortality; Amyotrophic Lateral Sclerosis - therapy; angiogenin; Angiotensinogen - administration & dosage; Angiotensinogen - adverse effects; Angiotensinogen - genetics; Biomedical and Life Sciences; Biomedicine; Cell Biology; Cells, Cultured; Female; gene therapy; Genetic Therapy - adverse effects; Genetic Therapy - methods; Genetic Vectors - administration & dosage; Genetic Vectors - adverse effects; Humans; Injections, Intramuscular; Internal Medicine; Laboratory Medicine; Male; Middle Aged; Pathology; Placebos; Transgenes - genetics; Treatment Outcome; Vascular Endothelial Growth Factor A - administration & dosage; Vascular Endothelial Growth Factor A - adverse effects; Vascular Endothelial Growth Factor A - genetics; VEGF; gene therapy; angiogenin; adenoviral vector; amyotrophic lateral sclerosis; VEGF
• ISSN: 0007-4888; 1573-8221
• DOI: 10.1007/s10517-008-0124-4

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.