New methods for CRISPR

A new study conducted at the UT Southwestern Medical Center was trying to use CRISPR on mice and human cells to correct a common mutation that underlies Duchenne muscular dystrophy (DMD). During the course of the study, researchers discovered that altering the dosages of the component sof the CRISPR improves how much dystrophin is produced. Dystrophin is the protein lost in DMD individuals which is essential for muscle control.

Normally, a 1 to 1 ratio of Cas 9 and guide RNA is used to guide a target to a certain part of the gene. However, the research group was not obtaining good results and thus decided to change this ratio. Through multiple trials, the researchers found that a 10 to 1 ratio of guide RNA to Cas9 resulted in optimal editing of the dystrophin gene, leading to eventual restoration of 90% of muscle function.

The research team is conducting a longer-term study using the same method to treat dogs with DMD. They hope that clinical trials are in store for the near future and hope to be able to extend this method to provide treatment for other childhood diseases.

Source
UT Southwestern Medical Center. "Scientists find method to boost CRISPR efficiency: Discovery made while editing genetic defect behind Duchenne muscular dystrophy." ScienceDaily. ScienceDaily, 6 March 2019. <www.sciencedaily.com/releases/2019/03/190306171244.htm>.

-Angela Wang