The very first study which would be testing the gene-editing technology CRISPR inside the body of a human is about to get underway in the US, according to news reports.
According to the Associated Press, the study intends to make use of CRISPR for the treatment of an inherited eye disorder which causes blindness.
Generally, people who suffer from this condition have a mutation in a gene which affects the functionality of the retina, which is the light-sensitive cells at the back of the eye which are necessary for normal vision. The condition is a type of Leber congenital amaurosis, one of the major causes of childhood blindness which affects around 2 to 3 newborns of every 100,000, according to the National Institutes of Health.
The mutation will be corrected by the treatment using CRISPR, which is a tool that enables researchers to precisely edit DNA in a certain spot, the AP reported.
Researchers will be using an injection for the delivery of the treatment directly to the light-sensitive cells, according to a statement from Editas Medicine, the firm which is conducting the study along with Allergan.
18 patients will be enrolled by the trial, both children (aged 3 and onwards) & adults.
The novel study is not similar to the controversial research of a Chinese scientist who employed CRISPR for editing the genomes of twin babies last year. In that case, the DNA of embryos was edited by the Chinese scientist, & these gene alterations had the possibility of passing down to the next generation, according to the AP report. In the novel study, the DNA edits made in the children & adults could not be passed down to their offspring, the AP told.