Genome Editing

Genome Editing
A team led by Katherine High at the Children Hospital of Philadelphia, a procedure known as ‘Genome Editing’ was performed. What they did is that they treated a life-threatening blood disease by repairing flaws in the genetic code of a living animal. It is first such feat achieved.
They treated mice that were bred to develop Haemophilia B, an inherited bleeding disorder in which the body’s ability to form blood clot is defective. About one in 30,000 boys are born with haemophilia B which in its most severe form may require frequent blood transfusions of Blood clotting factors to prevent spontaneous hemorrhages.
The work raised prospect of powerful new therapies that can target and repair the genetic defects behind a wide range of human diseases that cannot be treated by modern medicines.This new technique called genome editing holds particular promise for a diseases that run in families and are caused by faults in the genes that prevent healthy working of the immune system, bone marrow and liver.In the experimental mice who were suffering from Haemophilia B, the defect in the bleeding disorder was reversed to near normal without causing any apparent side effects.
Medical Tidbits 
Gene Therapy

 

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