Some medical conditions like beta-thalassemia, sickle disease among others, force people to live a life on blood transfusion. And the process can be exhausting for many.?
These diseases don¡¯t have a solution or a permanent cure that doesn't require regular blood transfusions, until now.
Two patients suffering from beta-thalassemia and one patient suffering from sickle cell disease no longer require blood transfusions thanks to?CRISPR.
In case you didn¡¯t know, CRISPR or Clustered Regularly Interspaced Short Palindromic Repeats is actually a powerful tool for editing genomes. It allows scientists to easily alter DNA sequences and modify gene function.
Here, they¡¯ve used it to treat severe forms of these inherited diseases by gene-editing bone marrow stem cells.
This trial was the first to use CRISPR to treat inherited genetic disorders. It was announced today at a virtual meeting conducted by the European Hematology Association.
Haydar Frangoul, one of the researchers at Sarah Cannon Research Institute in Nashville, Tennessee, said in a statement. ¡°The preliminary results¡ demonstrate, in essence, a functional cure for patients with beta thalassaemia and sickle cell disease.¡±
In case you didn¡¯t know, Beta thalassaemia and sickle cell diseases occur due to mutations affecting the haemoglobin, and as well all know, haemoglobin is responsible for carrying oxygen in red blood cells.?
Oddly enough, there are some cases where people with this disease don¡¯t show any symptoms. This is because their body doesn¡¯t stop producing fetal haemoglobin in adulthood -- something that stops its production after birth.
This discovery allowed scientists to develop a treatment based on boosting the performance of fetal haemoglobin. In the trial conducted by CRISPR Therapeutics and Vertex, boine marrow stem cells are extracted from people while the researchers disable the gene that turns off the production of fetal haemoglobin production with the help of CRISPR.
Remaining bone marrow cells are destroyed using chemotherapy, which is later replaced by edited cells. This makes sure new blood cells are produced by the edited stem cells.?
The treatment has some drawbacks, as chemotherapy can cause irreversible side effects like infertility among other issues. However, now the patients don¡¯t need blood transfusions anymore.