can turn off the chromosome responsible for Down syndrome
20.07. Warsaw (PAP) – It worked – at least in the laboratory – with a single gene to block operation of the additional chromosome, the presence of which is the cause of Down syndrome – informs “Nature”. Normal number of chromosomes contained in the cell, with which man is born to 23 couples, a total of 46 (two of these chromosomes are sex). In people with Down’s syndrome chromosome 47 – because chromosome 21 is present in them in two, but in triplicate.
extra chromosome causes symptoms such Down syndrome as a characteristic appearance, mental retardation (light to moderate), problems with the cardiovascular system and digestive tract or the early development of Alzheimer’s team.
Scientists
team Dr. Jeanne Lawrence (University of Massachusetts Medical School) was able to block the excess chromosome using XIST gene introduced into cultured in the laboratory of stem cells taken from people with Down syndrome.
XIST gene plays an important role in normal cell development in embryos of women – blocks the action of one of the two X chromosomes
authors of the study point out that the road to the practical use of these methods in the treatment of people with Down syndrome is far (talking about decades), but until recently, the very idea of ??blocking an entire chromosome gene therapy methods seemed unreal. So far, the researchers were able to barely (or not managed) to block a single defective gene (PAP)
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