US scientists try first gene editing in the body
Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease.
Nov 15, 2017
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Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease.
Nov 15, 2017
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1117
For most people with hemophilia B, whose bodies can't properly form blood clots, constant injections to replenish their clotting factors are a way of life. But now, Salk researchers have demonstrated in mice that hemophilia ...
May 1, 2018
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An international team of researchers has found more evidence that a new gene therapy may help some people with hemophilia A. In their paper published in the New England Journal of Medicine, the group describes the characteristics ...
Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated ...
Dec 11, 2011
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A team of researchers from institutions in Italy and the U.S., in conjunction with several corporate entities, has found that adding a protein to lentiviral vectors can protect them from an immune system attack. In their ...
In a study published today in the journal Nature Genetics, a group of scientists including UNC biologist Jason Lieb, PhD, present experiments supporting a longstanding hypothesis that explains how males can survive with only ...
Oct 24, 2011
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A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy treatment ...
Dec 6, 2017
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An innovative way to treat pediatric hemophilia A patients using a combination approach has seen early success at the Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, with researchers from Emory ...
Aug 29, 2019
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The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, ...
Jan 24, 2012
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A genetically engineered clotting factor that controlled hemophilia in an animal study offers a novel potential treatment for human hemophilia and a broad range of other bleeding problems.
Oct 23, 2011
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Haemophilia (English pronunciation: /hiːməˈfɪliə/; also spelled hemophilia in North America, from the Greek haima αἷμα 'blood' and philia φιλος 'love') is a group of hereditary genetic disorders that impair the body's ability to control blood clotting or coagulation, which is used to stop bleeding when a blood vessel is broken. Haemophilia A (clotting factor VIII deficiency) is the most common form of the disorder, present in about 1 in 5,000–10,000 male births. Haemophilia B (factor IX deficiency) occurs in around 1 in about 20,000–34,000 female births.
Like most recessive sex-linked, X chromosome disorders, haemophilia is more likely to occur in males than females. This is because females have two X chromosomes while males have only one, so the defective gene is guaranteed to manifest in any male who carries it. Because females have two X chromosomes and haemophilia is rare, the chance of a female having two defective copies of the gene is very low, so females are almost exclusively asymptomatic carriers of the disorder. Female carriers can inherit the defective gene from either their mother or father, or it may be a new mutation. Although it is not impossible for a female to have haemophilia, it is unusual: a female with Haemophilia A or B would have to be the daughter of both a male haemophiliac and a female carrier, while the non-sex-linked Haemophilia C, which can affect either sex, is extremely rare.
Haemophilia lowers blood plasma clotting factor levels of the coagulation factors needed for a normal clotting process. Thus when a blood vessel is injured, a temporary scab does form, but the missing coagulation factors prevent fibrin formation, which is necessary to maintain the blood clot. A haemophiliac does not bleed more intensely than a person without it, but can bleed for a much longer time. In severe haemophiliacs even a minor injury can result in blood loss lasting days or weeks, or even never healing completely. In areas such as the brain or inside joints, this can be fatal or permanently debilitating.
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