CRISPR, a groundbreaking and highly-controversial technique for gene-editing has been opted for use in a unique patient with some debilitating blood ailment as revealed by the scientists today. The Swiss and US companies behind this venture claim that this is the very first use of CRISPR on any human. This theory completely disregards the usage of this technique on China based cancer patients.
This unidentified patient registered for CRISPR clinical trial suffers from beta thalassemia which is a hereditary issue known to turn off one crucial gene that hampers our body’s ability to manufacture haemoglobin. The haemoglobin helps push oxygen all around the human body.
People suffering from beta thalassemia spend most of their lives depending on the blood transfusion process that helps offset this defect. However, the Swiss Biotech Company used the CRISPR therapy backed by the Vertex Pharmaceuticals at Boston and attempted to treat the disease with use of experimental DNA-editing to switch on the defective gene.
The firm confirmed that they will soon opt for the same treatment with one more patient who suffers from Sickle Cell Anemia, which is a blood-related issue that causes unbearable pain. With this stem-cell injection, the team inserts a good copy of the defective gene helping with perfect cure of the disease.