Scientists at the University of Texas in Austin have found a solution to tackle the off-centered effects of the gene editing system CRISPR-Cas9, a potential treatment for diseases caused by genetic aberrations. They suggest replacing Cas9 with a different protein, Cas12a.
Gene editing and gene therapy can delete disease-causing genes in order to treat specific diseases. But the skill to control unwanted genes is a point of concern, as it might cause severe side effects. A research team from Cardiff University and the University of Bath have devised a switch to control protein expression in mouse embryos, which could be used in human gene therapies in future.
A new risk assessment tool combining gene editing technology CRISPR-Cas9 with stem cells using disease modeling techniques has been developed by a team of scientists at the Stanford University Cardiovascular Institute. The tool has facilitated the modeling of individual patients’ heart in a lab and enabled undertaking of tests to ascertain whether or not signs of diseases are displayed by cardiac cells, as per a statement by the American Heart Association.
About half of all cases of deafness are caused by genetic defects. So it’s no surprise that the emerging gene-editing technique CRISPR-Cas9 is being examined closely for its potential to prevent hearing loss in people who inherit those genes. Scientists at the Howard Hughes Medical Institute are among those looking to deploy CRISPR in genetic deafness—and they have their first evidence that their technique may hold promise.
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