Developing Effective Therapy for Duchenne Muscular Dystrophy (DMD): Challenges and Promises

Title:Developing Effective Therapy for Duchenne Muscular Dystrophy (DMD): Challenges and Promises

Volume: 8 Issue: 3

Author(s): Trinath Ghosh, Ujjal Ghosh, Gargi Ghosh, Dabasish Malik, Shankhamala Bose and Utpal Basu

Affiliation:

Keywords: DMD, dystrophin, utrophin, myostatin, gene therapy, exon skipping.

Abstract: Duchenne Muscular Dystrophy (DMD) is one the most frequent genetic disorder that affects 1 in every 3500 males worldwide. This fatal neuromuscular disorder arises from the defects in the protein, called dystrophin. The dystrophin coding gene is the largest known gene and present in X chromosome. Several strategies, ranging from cell based therapy to small RNA mediated exon skipping have been proposed as an effective therapy for this disease. Experiments in mice model have shown that upregulation of utrophin, the autosomal homologue of dystrophin can compensate dystrophin deficiency and ameliorate the dystrophic phenotype. Therefore utrophin has also been considered as a potent target for development of strategies against DMD. In the current review we describe different therapeutic approaches for DMD along with challenges they have to overcome.

Cite this article as:

Ghosh Trinath, Ghosh Ujjal, Ghosh Gargi, Malik Dabasish, Bose Shankhamala and Basu Utpal, Developing Effective Therapy for Duchenne Muscular Dystrophy (DMD): Challenges and Promises, Current Chemical Biology 2014; 8 (3) . https://dx.doi.org/10.2174/221279680803150420094222