Since long time Scientist is working on CRISPR Gene-editing technology may be a powerful “molecular scissors.” Researchers in the US have been working CRISPR/Cas9, it can edit genes in an adult who are having genetic diseases such as sarcoma, sickle cell disease, and beta-thalassemia. The doctor very soon comes to know how well it will work on people.
CRISPR/Cas9 cuts through DNA. But it doesn’t randomly chop. This gene editor comes in two parts. The CRISPR part is a short piece of genetic material called a guide RNA. Like seeing-eye dogs, these guides lead Cas9, an enzyme, to where it needs to go. Scientists can use CRISPR/Cas9 to intentionally break some genes or repair others.
CRISPR Gene-editing technology first CRISPR trial in Europe and the US, which enrolled its first patient in February this year, aims to treat beta-thalassemia and sickle cell disease, two blood disorders that affect oxygen transport in the blood. The therapy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals, consists of harvesting bone marrow stem cells from the patient and using CRISPR technology to make them produce fetal hemoglobin, a natural form of the oxygen-carrying protein that binds oxygen much better than the adult form.
Before the trial started, the FDA put it on hold in the US to clear out some safety questions. A few months later, the hold was lifted and the treatment was given fast track designation for both conditions.
CRISPR is a great candidate to treat genetic blindness. Many hereditary forms of blindness are caused by a specific mutation, making it easy to instruct CRISPR-Cas9 to target and modify a single gene
The first applications of CRISPR could be in cancer. Indeed, one of the first and most advanced CRISPR clinical trials, which is currently running in China, is testing the potential of the gene-editing tool to treat patients with advanced cancer of the esophagus.
There are several ways CRISPR technology could help us in the fight against AIDS. One is using CRISPR to cut the DNA of the HIV virus out of its hiding place in the DNA of immune cells. This approach could be used to attack the virus in its hidden, inactive form, which is what makes it impossible for most therapies to completely get rid of the virus.
It’s difficult to say CRISPR will solve everything, challenges still remain but only time will tell problems can be solved or not.
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