Extra Chromosome Silenced in Down Syndrome Cells

Amazing news was announced in the field of gene therapy this week. Scientists in Massachusetts have taken the cells of a person with Down Syndrome and have silenced the extra 21st chromosome in those cells. The Guardian has the story:Scientists have corrected the genetic fault that causes Down's syndrome – albeit in isolated cells – raising the prospect of a radical therapy for the disorder.In an elegant series of experiments, US researchers took cells from people with DS and silenced the extra chromosome that causes the condition. A treatment based on the work remains a distant hope, but scientists in the field said the feat was the first major step towards a "chromosome therapy" for Down's syndrome."This is a real technical breakthrough. It opens up whole new avenues of research," said Elizabeth Fisher, professor of neurogenetics at UCL, who was not involved in the study. "This is really the first sniff we've had of anything to do with gene therapy for Down's syndrome."The scientists used a gene normally found on the X chromosome to shut down the extra chromosome. Men have one X chromosome in their cells, and women have two. Only one X chromosome is needed for a cell to function, so the Xist gene inactivates one of the X chromosomes by covering it in RNA. Researchers were able to insert the Xist gene in the chromosome 21, which then silenced it.This breakthrough could also be applied to other disorders like Edward syndrome which is trisomy 18...
Source: Mary Meets Dolly - Category: Geneticists and Genetics Commentators Tags: Genetic Engineering Source Type: blogs