New CRISPR/Cas9 Technique Treats Cystic Fibrosis In Human Stem Cells

Aug, 2021 - by CMI


Researchers are revamping genetic mutations that can cause cystic fibrosis in enlarged human stem cells by a process called prime editing to replace with a healthy piece of DNA

Cystic fibrosis (CF) is one of the most common genetic diseases worldwide and has serious side effects for patients. The tissues of the lungs and intestines are sticky and thick, causing congestion. Although therapies are accessible to reduce mucus and prevent inflammation, CF is still not curable. Now, a new study from a group of scientists at the Hubrecht Institute in partnership with UMC (Utrecht and the Oncode Institute) offers new hope.

Researchers were successful in correcting mutations that cause CF in human organoids. They were previously formed by the same research team from stem cells of patients with CF and stored at a biobank in Utrecht. A procedure termed as prime editing was used to replace the altered DNA fragment that causes CF with a healthy strand of DNA in these organoids. Advanced editing new version of CRISPR / Cas9. CRISPR / Cas9 cutbacks the DNA before processing it. Although this repairs a modified DNA fragment, it also damages the other parts of the genome. Studies prove that the new technique is safer than the standard CRISPR / Cas9. It can form on a new part of DNA without affecting elsewhere in the DNA. As a result, CF mutations were found in the CFTR channel, which is present in cells of various structures including the lungs. As a result of the mutations, the channel does not function properly, leaving a layer of mucus covering the cells with very little water. Currently, the researchers have shown that changes in CF are safe to address the challenges by clinically applying them into the stem cells. New CRISPR / Cas9 variants, such as key programming can safely modify changes without causing damage to other DNA regions. Hopefully, this will enable doctors to treat and prevent genetic diseases in the future.