Sunday, December 11, 2016

Breakthrough in Gene Therapy for Sickle Cell Disease

Breakthrough in Gene Therapy for Sickle Cell Disease

A team of researchers are making breakthroughs when it comes to correcting defective sickle cells with a gene-editing tool called CRISPR. This tool can fix genes that cause sickle cell, and thus can lead to promising gene therapies for this ailment.

The researchers have been able to prove that they can use such mended cells to make a “high functioning hemoglobin molecule” and have it transfer oxygen to cells. When the stem cells were placed in mice they found it successful in treating disorders such as sickle disease and thalassemia.
CRISPR is both an enzyme and “guide RNA” that can cut the part of the gene that causes mutation out and use other tools to make the correct sequence.

Sickle Cell is a disease that make normal cells turn into a sickle shape, which significantly decrease the amount the oxygen being transported around the body. This new breakthrough with CRISPR can be used to help change treatment, and detect and prevent disease in those who fall ill.


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  1. This would be huge to help anyone affected with sickle cell and would better the world in the future if the CRISPR gene tool actually works! Only hoping it does work for such a terrible disease that is out in the world.

  2. This article provides excellent information about CRISPR. It is quite daunting to think about the amount of people being affected by the sickle cell disease everyday. However, this is a breakthrough discovery which can help millions of people.