Image copyright Thinkstock The British and Swedish scientists announced the start of clinical trials for the introduction of embryonic stem cells.
They hope that through this method it will be possible to prevent or significantly reduce the probability of the children of a number of hereditary diseases, such as osteogenesis imperfecta, characterized by increased fragility of bones.
The tests will begin in January in the Swedish Karolinska Institute and Children’s Hospital at Great Ormond Street in London.
Scientists use stem cells obtained from terminated pregnancies.
Fragile Children
Osteogenesis imperfecta (osteogenesis imperfecta) is shown at one of the 25 thousand newborn.
Babies are born with multiple fractures and often do not survive. At the same ones who survived, there are problems of development, hearing, dental problems, and each year they happen to 15 bone fractures.
This is because of violations of the DNA at the stage of fetal development. These children collagen – fibrillar protein that forms the basis of connective tissue – or of very poor quality, or even completely absent.
Professor Lyn Chitty from the hospital on Great Ormond Street will hold a series of genetic tests to identify the cause of the such disease.
“This is a very serious disease. Our goal – to understand whether stem cells to prevent its development in the womb,” – she said in an interview with BBC BBC.
embryos prerequisites to the development of osteogenesis imperfecta will be introduced form of stem cells that can transform into cartilage, bone and muscle tissue.
15 babies the procedure will be performed on the stage of development in the womb and immediately after birth, and another 15 – just after birth. Families participating in the experiment will be monitored for two years.
The first group of cells will be administered between 20 and 34 weeks of pregnancy. At this stage of the sexual organs of the embryo is already formed, and the risk of damage to the reproductive system of the fetus is not.
The probability of rejection of the fruit of extraneous tissue, scientists say very little.
The story of Adam
21-year-old Briton, Adam Reynolds from Hampshire was born with broken arms and cracked spine. According to him, the main problem as a child was that I had all the time myself to stop because “children want all the time to rush and play with their peers”.
When he played football, then mostly stood at the gate, in order to reduce the risk of fractures.
According to Adam, for his life, he broke the bones of about 40 times. This is less than many other patients with osteogenesis imperfecta, but his injuries heal more slowly than others.
In May 2009, he broke his leg. Since it was six years old, he underwent 12 operations, but a full recovery to achieve and failed. His left leg 10 centimeters shorter than the right.
“Life is, of course, no sugar. If an ordinary person will hook the little finger of the leg of the table, he just says,” Oh. “I exclaim:” Again broke! “- divided Adam.
However, he does not believe that his illness was the obstacle in life. Last week he received his first degree in “Accounting and Finance” and has already started to work.
“That might have come up with a way to combat this phenomenon, it’s just grand,” – says Adam.
musculoskeletal horizons
At this step on to fully defeat the disease, it does not go. According to scientists, if using stem cells, they will learn to strengthen bones to at least reduce the number of cracks and fractures in people with a similar diagnosis and improve the mechanisms of skeletal development, it has become a great achievement.
The main condition for success – to begin treatment before the bones were formed and began to develop, that is, the stage of pregnancy.
At the same time, scientists have noted that the results the treatment will largely depend on the form of the disease, and thus stem therapy is unlikely to be equally effective for everyone.
Implantation of stem cells have already been used in two cases, after identifying the fetus signs of bone formation, but no large-scale clinical trials talk about the effectiveness of therapy is not possible.
“Such tests on embryos for the first time, and if all goes well, it will open horizons for the treatment of various kinds of abnormalities in embryos when parents will have no alternative,” – said Dr. Cecilia Goterstrom of the Karolinska Institute in Sweden.
She said that one day stem cells play a role in the prevention and treatment of Duchenne muscular dystrophy and other diseases of the muscular system.
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