Recently, a private biotech company has declared that it combined the bacteria-derived gene-editing tool, CRISPR, with stem cell therapy so as to help cure a human patient dealing with blood-related disorders. It is the first of its kind breakthrough that has been made developed outside of China. The companies named Vertex Pharmaceuticals and CRISPR Therapeutics are working on developing a cure for beta thalassemia by using the CRISPR/Cas9 hematopoietic stem cell therapy or CTX001.
It new technique is currently in the early days and still has to pass the safety and efficacy phases before given a green card to be used in humans. It is also been used to test if the severe sickle cell disease can be also be treated. By the mid of 2019, the US will have a human trial phase commencing. The companies and researchers believe that the CTX001 has come a long way and it is for the first time going to be tested in a beta-thalassemia patient in a clinical study. This will be the first milestone and the beginning of a realization that CRISPR/Cas9 therapies need to be placed in a new class owing to their transformative nature in terms of treating serious diseases. The beta-thalassemia patients have their fetal hemoglobin in red blood cells increased using CRISPR/Cas9 technology.
The fetal hemoglobin is present right from the birth and hence there can be no changes or replacement brought in it but manipulating the genetic information to transform into the existing fetal gene is being worked on. Even the manipulation or editing of the germline cells including the sperms and eggs that will bring about inheritable changes in every cell in the body is also impossible. The blood cells possessing the ability to hold more of oxygen and boost the levels of fetal hemoglobin production are being reinfused in the patient’s bloodstreams. This can prove to become a one-time potential curative therapy as this transformative medicine can help treat serious diseases like beta-thalassemia and sickle cell disease. A neuron-optimized CRISPR activation system created by Jeremy Day and colleagues have helped study the use of CRISPR/Cas9 genome editing technology in order to regulate genes in the brain. This method helps open new doors for researchers studying genetic influences on brain health or other diseases. Though it is in the clinical stage it is definitely going to clear the human trial as well.