Due to the ‘cut and paste’ gene-editing technique CRISPR, scientists are homing in on a treatment for sickle cell disease—a genetic blood disorder—whereas different research is how to broaden the potential of CRISPR-based treatments.
Earlier this decade, geneticists discovered that they might easily re-program bacterial immune machinery to create a gene-editing tool that is ready to search for genes in plant and animal cells. They might also manipulate the enzyme known as Cas9 used in bacteria in order to insert new DNA into genes.
The CRISPR-Cas9 gene-editing method was born, and what was months of lab work to edit genes turned to days.
Since then, speculation has been rife in regard to the potential applications of fast and simple gene editing. Aside from guarantees of sterile mosquitos and disease-resistant bananas, there have been claims that CRISPR might delete disease-causing genes, or add in ‘healthier’ variations.
At the Institut Imagine in Paris, France, scientists are utilizing CRISPR to see if they’ll treat a genetic disorder generally known as sickle cell disease
In sickle cell disease, stem cells within the bone marrow can suffer a mutation within the gene responsible for hemoglobin, the protein in blood that carries oxygen. The result is withered, hardened red blood cells that carry oxygen poorly.
The immune response is likely one of the major barriers for scientists who want to deliver CRISPR-based mostly treatments into patients’ cells. These ‘gene therapies’ can be used to treat cells in a lab or directly within the patient’s body.