Gene Editing: CRISPR Cas 9 News

Gene Editing

Genetic engineering has improved in the past decade. The scientific community is working on perfecting CRISPR Cas 9 gene editing. An article published by CNN Health News describes how scientists edit disease-causing gene mutation. I have summed up the main points in this blog post.

For the first time, scientists said, they corrected a gene mutation linked to inherited heart conditions in human embryos using the approach. A study demonstrating the technique was published in the journal Nature on Wednesday (PDF).

“This is the first that has been demonstrated as safe and working,” said Belmonte, a professor at the Salk Institute for Biological Studies’ gene expression laboratory in La Jolla, California.

“All cells of the embryo were corrected,” he said. “It seems to be working from these samples that we have chosen, but we need to do much more basic research with many other genes.”

Scientists estimate that more than 10,000 human diseases may result from mutations to a single gene occurring in all cells of the body, according to the World Health Organization.

The goal was to correct a type of inherited heart condition. A mutation called MYBPC3 is associated with inherited heart conditions, including left ventricular noncompaction, familial dilated cardiomyopathy and familial hypertrophic cardiomyopathy, which affects an estimated one in 500 people worldwide.

In the far-off future, a clinical trial could include transplanting corrected embryos into a uterus with the goal of establishing pregnancy and then monitoring the embryos as they develop into children.

However, the researchers wrote in their study that they hope CRISPR could be considered as an alternative option to preimplantation genetic diagnosis, also known as PGD, for couples at risk of passing on an inherited disease.