World first: Stem cell treatment for lethal STAT1 gene mutation shows 'disappointing,' but promising results
One instance of STAT1 GOF Mutation phenotype. Credit score: Picture courtesy of Hiroshima College The primary ever examine assessing how sufferers with "achieve of operate" mutation of the STAT1 gene reply to stem cell transplantation has taken place. It concerned 15 younger sufferers, from 9 totally different international locations, every struggling a spread of issues attributable to the gene's mutation. Of those, solely six survived a regime of stem cell transplantation -- with 5 utterly cured and illness free by the examine's conclusion. The examine was carried out by Dr. Satoshi Okada (Hiroshima College), Professor Jennifer Leiding (College of Florida), Professor Tomohiro Morio (Tokyo Medical and Dental College), and Professor Troy Torgerson (College of Washington). Dr. Okada, who first found the STAT1 achieve of operate mutation in 2011, says that "General, this result's disappointing -- however the truth 5 suffe...