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In search of a cure for genetic disorders

By Bindu Shajan Perappadan

NEW DELHI, NOV. 10. Geneticist Michel Sadelain is among the front-runners in the "research industry" of gene transfers and he could well be on his way to finally giving the world a cure for ailments previously considered incurable.

Genetic disorders such as sickle cell anaemia, cancer and thalassaemia could be on their way out if what this geneticist is trying works. What Dr. Sadelain is trying to do is treat diseases by inserting a healthy gene in place of one that is malfunctioning or missing. Simple as it might sound, the research has taken him over 12 years and has finally been cleared for trials on large animals.

The researcher was in town to participate in the Third International Conference on Thalassaemia organised by the Thalassaemia India and Sir Ganga Ram Hospital in collaboration with Thalassaemia International Federation here this past week. He has been doing ground-breaking work in the area of gene transfer, trying to insert genes into bone marrow cells and T-lymphocyte (a type of white blood cell) to reduce the risk of graft-verus-host disease, a common and potentially dangerous complication of bone marrow transplant, while preserving the anti-leukaemia effect of the bone marrow transplantation.

Another of Dr. Sadelain's goals is to enhance the use of T-lymphocytes as therapeutic tools by targeting the cancer cells, making sure that they go where they should in the body to kill tumour cells, including melanoma and prostate cancer cells.

He is also seeking to improve gene transfer and gene expression in blood forming cells. His ultimate goal, however, is to use gene transfer mediated by retroviruses to create improved treatment for genetic disorders, such as sickle cell anaemia and cancer.

"Many people are working in the area, but we believe that we could well be on the way to giving the world a cure and though all of this might sound very simple it has taken us a while to figure this one out. Also, what has taken us a long time to do is work at vector which will take the gene replacement to the target site," explained Dr. Sadelain.

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