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.
Sources:
1 1. https://www.sciencedaily.com/releases/2016/11/161108112133.htm#
