Spinal muscular atrophy disease: a literature review for therapeutic strategies

• Published in: Journal of medicine and life Vol. 3; no. 1; pp. 3 - 9
• Main Authors: Stavarachi, M, Apostol, P, Toma, M, Cimponeriu, D, Gavrila, L
• Format: Journal Article
• Language: English
• Published: Romania Carol Daila University Foundation 2010; Carol Davila University Press
• Subjects: Anabolic Agents - therapeutic use; Exons; Genetic Therapy; Humans; Muscular Atrophy, Spinal - genetics; Muscular Atrophy, Spinal - therapy; Neuroprotective Agents - therapeutic use; Promoter Regions, Genetic; Review; Stem Cell Transplantation; Survival of Motor Neuron 2 Protein - genetics

Currently, there is no cure for the treatment of spinal muscular atrophy (SMA). Based on the available clinical and molecular findings, different therapeutic strategies were tested in vitro and in vivo and clinical trials are ongoing. The main therapeutic direction is focused on the enhancement of S...