Research
Lab's Research Interest
Our lab specializes in Adeno-Associated virus (AAV) vector mediated gene therapy for multiple genetic disorders. Our special interest is in the engineering of AAV vectors for efficient and safer gene delivery. The focus of our lab is mainly on translating novel AAV based therapeutics from bench to bedside. Recently, our lab has licensed multiple gene therapies, paving the way for more afforadable gene therapy treatments.
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.
