Spinal Muscular Atrophy Disease: a Literature Review for Therapeutic Strategies

Overview

Journal J Med Life

Specialty General Medicine

Date 2010 Mar 23

PMID 20302191

Citations 13

Authors

M Stavarachi

P Apostol

M Toma

D Cimponeriu

L Gavrila

Affiliations

Soon will be listed here.

Abstract

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 SMN expression by increasing the full-length (fl) SMN2 transcript levels, preventing the SMN exon 7 from skipping or from protein stabilizing. In addition, the action of neurotrophic, neuroprotective or anabolic agents is tested and stem cell and gene therapy approaches are in a promising development.

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