Spinal muscular atrophy disease: a literature review for therapeutic strategies
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Updated: Jun 14, 2026

Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice
Published on: October 3, 2011
M Stavarachi1, P Apostol, M Toma
1Human Genetics and Molecular Diagnosis Department, Institute of Genetics, 1-3 Portocalelor Street, District 6, postal code 060101, Bucharest, Romania. monica.stavarachi@gmail.com
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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