Immunological and Autoimmune Disorders

Gene editing, a possible treatment, for inherited kidney disease Joubert syndrome, an inherited brain disorder that causes kidney failure in patients. This requires a transplant or dialysis. A mutation in the gene CEP290 causes the inherited form of kidney disease and scientists in the U.K. to apprise of having an early proof of gene editing called “exon skipping” that can correct the error. The researchers at Newcastle University used a synthetic strand of DNA to deceive cells into bypassing the damaging CEP290 mutation. They used kidney cells taken from a 19-year-old patient with Joubert syndrome for their early testing of the technique. The New England Journal of Me...