Media has a way of portraying science fiction and scientific technologies in an outrageous way, like changing humans into creatures through the use of CRISPR (clustered regularly interspaced short palindromic repeats) technology. CRISPR was seen in bacterial defense and was used to destroy DNA that entered the bacteria, but is now used to edit and alter specific DNA sequences. In humans, though, CRISPR can ultimately be used to treat improve health, improve food, treat cancers and diseases and potentially suppress the effect of environmental pollution.
How CRISPR works.
However, concerns are arising on the problems with CRISPR. Some research suggests that the use of CRISPR at a specific sequence can damage DNA further along the chromosome, and this makes some caution to be needed when using CRISPR in clinical settings.
In humans, some trials have been planned to help treat human diseases. Two main trials have been planned in order to treat blood diseases (beta-thalassemia and sickle cell anemia). The trials use gene editing outside of the patient’s body (ex vivo). The thought is that researchers will take hematopoietic blood cells and edit them and “correct” them. Once the genes are corrected and the cells are normal, they will be reintroduced into the patient’s body to cure the disease. This theory has been used in China to test treatments on human cancers. Using immune T cells, researchers use CRISPR to stop the cells from producing a PD-1 protein, and allows T cells to now attack cancer cells.
Although in theory these treatments seem flawless, there are some concerns. Some studies suggest that using CRISPR may not be as effective as thought and have some side effects. One study says that when Cas9 cuts the DNA, it can potentially cause it to not be able to edit the DNA anymore. A bigger concern is about human exposure to CRISPR. Since CRISPR is a protein found in bacteria, many individuals may have already been exposed to it from bacterial infections, which causes the body to develop an immune response to the protein and therefore are unable to benefit from the CRISPR therapies. The last concern is that CRISPR is not totally accurate and may have the potential to cut the wrong DNA sites and cause disease.