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It is caused by mutations in the survival motor neuron 1 (<jats:italic><jats:styled-content style=\"fixed-case\">SMN<\/jats:styled-content>1<\/jats:italic>) gene, leading to the reduction of <jats:styled-content style=\"fixed-case\">SMN<\/jats:styled-content> protein. The most striking component is the loss of alpha motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment other than supportive care, although the past decade has seen a striking advancement in understanding of both <jats:styled-content style=\"fixed-case\">SMA<\/jats:styled-content> genetics and molecular mechanisms. A variety of disease modifying interventions are rapidly bridging the translational gap from the laboratory to clinical trials. In this review, we would like to outline the most interesting therapeutic strategies that are currently developing, which are represented by molecular, gene and stem cell\u2010mediated approaches for the treatment of <jats:styled-content style=\"fixed-case\">SMA<\/jats:styled-content>.<\/jats:p>","DOI":"10.1111\/jcmm.12224","type":"journal-article","created":{"date-parts":[[2014,1,9]],"date-time":"2014-01-09T02:04:55Z","timestamp":1389233095000},"page":"187-196","source":"Crossref","is-referenced-by-count":23,"title":["Molecular, genetic and stem cell\u2010mediated therapeutic strategies for spinal muscular atrophy (<scp>SMA<\/scp>)"],"prefix":"10.1111","volume":"18","author":[{"given":"Chiara","family":"Zanetta","sequence":"first","affiliation":[{"name":"Dino Ferrari Centre Neuroscience Section Department of Pathophysiology and Transplantation (DEPT) University of Milan Neurology Unit IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico Milan 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