Gene Therapy

A major thrust of the center has been on developing gene therapy towards clinical trials. Two areas are being developed in collaboration with different groups - the first is the gene therapy program for hemophilia using AAV vectors and the second gene therapy program is towards the major hemoglobin disorders. A clinical trial for gene therapy of hemophilia B is proposed and will be initiated shortly.

Musculoskeletal Regeneration

The emphasis of this theme is on regeneration of musculoskeletal tissues such as cartilage (physeal and articular cartilage), bone and muscle using stem cells and differentiated cells. Major areas of research are cartilage tissue engineering for treatment of physeal and articular cartilage defect. A clinical trial in currently ongoing with the aim is to effectively translate musculoskeletal stem cells and differentiated cells for commonly encountered clinical problems which have less than satisfactory available treatment.

Cellular Reprogramming and its Applications – Haplobanking and Disease Modeling

CSCR is also engaged with the creation of an induced pluripotent stem cell “haplobank”. iPSCs derived from individuals with homozygous HLA haplotype will be banked for future research and potential therapeutic applications. iPSC models for some rare diseases are created to study the disease biology and explore novel therapeutic approaches.