Dr Parvathy Venugopal
The need
Inherited bone marrow failure (BMF) disorders are conditions where the bone marrow can't produce enough blood cells, often affecting children and leading to a higher risk of blood cancers. The only long-term solution for severe cases is a stem cell transplant from a donor. However, finding a good match is challenging, as the best donors are often family members who may carry the same genetic issues.
The research
Dr Venugopal's research explores somatic genetic rescue often known as “natural gene therapy”, where a natural correction occurs in some of the blood cells, leading to a partial or full recovery from BMF. She aims to induce this natural process in a faithful disease model to help develop a novel treatment for human BMF.
The impact
This research aims to help patients with BMF regain healthy bone marrow function thereby reducing their risk of developing blood cancer. This approach seeks to provide a safer alternative to the current standard of care which is accompanied by significant risks including death.
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