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The AAV Vector as a SMA Disease Modifier

Ana Cristina Martins De Figueiredo

The discovery of DNA as the biomolecule of genetic inheritance and disease opened the prospect of therapies in which mutant and damaged genes could be altered for the improvement of the human condition.1,2 Genome-editing is a technology that allows specific changes in the genes of interest. This approach is capable of manipulating the genome of living cells or organisms in various ways: insertions or deletions of chosen genes, introduction of point mutations, knockout or correction of specific genes3 Gene therapy provides a unique approach to treat a variety of both inherited and acquired diseases2 by delivering a therapeutic gene material to correct the loss‐of‐ function caused by mutation or to express the deficient gene product.