Research

My Research Focus

Recurrent and spontaneous bleeding occurs in large joints in hemophilic patients, leading to joint impairment, which is a major comorbidity in hemophilic patients and is termed hemophilic arthropathy. The pathophysiology includes chronic inflammation of joints, cartilage deterioration, and synovial hypertrophy, but chronic inflammation is the hallmark condition that deteriorates the disease.  Duchenne muscular dystrophy is yet another X linked genetic disorder caused by a mutation in the dystrophin gene. The dystrophin glycoprotein complex is compromised in DMD, due to which normal skeletal muscle contraction leads to mechanical stress induced muscle injury. This leads to a chronic inflammatory response, exacerbating muscle fiber damage and turnover. Inflammation being the root cause of the worsening of both diseases, my research will primarily focus on targeting the pro-inflammatory molecules and regulating their expression by using microRNAs delivered through adeno-associated virus vectors. This therapy will ameliorate the inflammation in the hemophilic joint in HA and muscle fiber turnover in DMD, leading to a reversal of the disease phenotype.