After six years of work, that experimental treatment has now been approved for clinical trials by the U.S. Food and Drug Administration, enabling the first tests in humans of a CRISPR-based therapy to directly correct the mutation in the beta-globin gene responsible forsickle cell disease. Bet...
Gene-editing techniques such as CRISPR/Cas9 can beused to astonishing, potential life-altering effect, such as one demonstration involving a possible treatment for ALS. Researchers at Carnegie Mellon University and Yale University have now made a significant breakthrough in the technology’s usefulness...
2012). What happens next is highly dependent on how the break is repaired by the cell, which can vary depending on factors such as the cell type/state and availability of a repair template (reviewed in Ceccaldi et al. 2016). Precise changes can be introduced into the genome using CRISPR/...
On November 15th, the scientific world was taken by storm when Chinese researchers announced that the gene editing technique, CRISPR, was being used for the first time in a clinical trial.i CRISPR, which stands for clustered regularly interspaced short palindromic repeats, allows scientists to targe...
Gene drugs will focus on the causes of diseases rather than symptoms and doctors will start to prescribe different treatment plans for different patients according to the genetic characteristics of patients. Many potential diseases will be cured before the diseases appear, but cracking the book of ...
“Adult-onset genetic disorders haven’t received as much research attention as those that appear in early life,” she says. “By finding more people with these rare adult-onset diseases, and those who may be pre-symptomatic, we’ll be able to learn more about a whole range of rare di...