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Showing posts from October, 2016

Autophagy: A Nobel Prize Topic

        Introduction The word autophagy is derived from Greek words “auto” meaning self and “phagy” meaning eating. Autophagy is a normal physiological process in the body that deals with destruction of cells in the body. It maintains homeostasis or normal functioning by protein degradation and turnover of the destroyed cell organelles for new cell formation. During cellular stress the process of Autophagy is upscaled and increased. Cellular stress is caused when there is deprivation of nutrients and/or growth factors. Thus Autophagy may provide an alternate source of intracellular building blocks and substrates that may generate energy to enable continuous cell survival. History Autophagy has been known for over 50 years but its fundamental importance in physiology and medicine was only recognized after Yoshinori Ohsumi's paradigm-shifting research in the 1990's. For his discoveries, he is awarded this year's Nobel Prize in physiology or medicine.

Promising Gene Therapy For Sicker Cell Anemia

A new engineered gene therapy virus, inserted into blood stem cells and then transplanted into mice with sickle cell disease, markedly reduced red blood cell damage according to the study “Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype,” published in the Journal of Clinical Investigation. A clinical gene therapy trial is expected in the coming year in which researchers will use a gene manipulated harmless virus to prevent the “sickling” of red blood cells. The new gene therapy is based on research going back to the 1980s which revealed that people with a milder form of sickle cell disease carried a fetal form of hemoglobin. This form is present in the human fetus and normally tapers off after birth. It differs most from “adult” (beta) hemoglobin because it is able to bind oxygen to a larger extent and is not seen to “sickle”. In later studies, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center researchers showed t