Mir-196A: Is It the ‘silver Bullet‘ Against Huntington‘s Disease?

Abstract

EVALUATION OF: Cheng PH, Li CL, Chang YF et al. miR-196a ameliorates phenotypes of Huntington diseases in cell, transgenic mouse, and induced pluripotent stem cell models. Am. J. Hum. Genet. 93(2), 306–312 (2013). This study reports on the therapeutic potential of miR-196a in alleviating the molecular, pathological and behavioral deficits associated with Huntington‘s disease in several model systems, often used in the study of the disease. miR-196a appears to confer a robust neuroprotective role through the targeting of molecules that are downstream of the neurodegenerative cascade and part of the ubiquitin proteosome, gliosis and CREB pathways. miR-196a, however, does not appear to target HTT or mutHTT. Owing to the far-reaching role of the miRNA, it may be considered relevant in the treatment options of other brain diseases and disorders characterized by the misfolding and aggregation of host proteins of the CNS.

KEYWORDS:

• Huntington‘s disease
• miRNA
• mouse model
• neurodegeneration
• neuroprotection
• proteinopathies

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.