Sickle-Cell Anaemia:First Time cured by Gene Therapy

Date: March 07, 2017

Sickle-cell disease (SCD) haemoglobin sickle-cell crisis

Sickle-cell disease (SCD) is a group of blood disorders typically inherited from a person's parents.The most common type is known as sickle-cell anaemia (SCA). It results in an abnormality in the oxygen-carrying protein haemoglobin (hemoglobin S) found in red blood cells. This leads to a rigid, sickle-like shape under certain circumstances.Problems in sickle cell disease typically begin around 5 to 6 months of age. A number of health problems may develop, such as attacks of pain ("sickle-cell crisis"), anemia, swelling in the hands and feet, bacterial infections, and stroke.Long term pain may develop as people get older.


Why in News:-

A Teenage boy with an inherited disease that affects millions worldwide seems to have been cured using gene therapy.The treatment appears to have stopped the painful symptoms of sickle cell disease, demonstrating the potential for gene therapy to treat common genetic diseases.

The idea of gene therapy – using strands of DNA to compensate for a person’s malfunctioning genes – is almost three decades old.However, the approach has so far mostly been used to treat very rare diseasesIn contrast, sickle cell disease affects 100,000 people in the US alone.