Muscular dystrophy is inherited in an X- linked recessive pattern. There are two types of muscular dystrophy; Duchenne muscular dystrophy (DMD); which patients generally die in their twenties, and Becker muscular dystrophy (BMD); which patients show milder symptoms and the disease progresses more slowly. Muscular dystrophy is caused by a loss of functional mutation in DMD gene which codes for a structural protein called dystrophin. Dystrophin supports fiber strength, therefore its absence leads to stiffness reduction and progressive muscle weakness. Although numerous approaches such as medications, physical therapy and surgery have been used for decades to treat muscular dystrophy, they have not been efficient enough and suffer from a variety of drawbacks. Stem cell therapy for muscular dystrophy can potentially bring improvement and have had promising successes in dystrophic mouse models. Only a small number of cells together with stimulatory signal for expansion is required to obtain a therapeutic effect. Beside stem cells, other therapeutic strategies such as gene therapy are being tested in patients and pre-clinical models. Since almost all types of muscular dystrophies arise from single-gene mutations, gene therapy, involving replacement or modification of a gene, has emerged as a promising approach for treatment. Three relatively new gene therapy strategies are: the delivery of functional mini- and micro-dystrophins by recombinant adeno-associated viral (rAAV) vectors, exon skipping and dystrophin replacement by utrophin upregulation. These three novel methods have several advantages compared with other approaches which mark them as effective strategies for DMD treatment. Steady progress in understanding the DMD gene and its function has resulted in several innovative therapeutic approaches. So far, the antisense approach seems the most promising treatment for clinical trials due to its efficiency and simplicity. It is expected that future studies bring great advances in this field.

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