Posts Tagged: death

World first: Stem cell treatment for lethal STAT1 gene mutation — shows ‘disappointing’ but promising results

World first: Stem cell treatment for lethal STAT1 gene mutation — shows ‘disappointing’ but promising results

The first ever study assessing how patients with “gain of function” mutation of the STAT1 gene respond to stem cell transplantation has taken place. It involved 15 young patients, from nine different countries, each suffering a range of complications caused by the gene’s mutation. Of these, only six survived a regime of stem cell transplantation – with five completely cured and disease free by the study’s conclusion.   The study was carried out by Dr. Satoshi Okada (Hiroshima University), Professor Jennifer Leiding (University of Florida), Professor Tomohiro Morio (Tokyo Medical and Dental University), and Professor Troy Torgerson¬† (University of Washington). Dr. Okada, who first discovered the STAT1 gain of function… Read more