Sentences with word «thalassaemia»

In humans, this can lead to genetic blood disorders such as thalassaemia, and possibly cancer.
A subtle form of gene therapy for thalassaemia has received a boost with the creation of a modified virus that successfully shuttles the required genetic machinery into cells.
In one of the forms of thalassaemia they studied, correctly spliced products rose from 10 per cent to more than 80 per cent (Proceedings of the National Academy of Sciences, vol 90, p 8673).
One form causes thalassaemia in Greek people and one in Chinese people; the other causes a rare form of the disease in Mediterranean people.
Al Matrooshi was born with an inherited blood condition called beta thalassaemia which can be fatal if left untreated.
A 21 - YEAR - OLD Frenchman is the first person in the world to be cured of the blood disorder beta - thalassaemia through gene therapy.
People with thalassaemia or sickle cell anemia have damaged adult haemoglobin — the vital molecule that picks up oxygen in the lungs and transports it around the body — and they require life - long treatment with blood transfusions and medication.
L1 has been widely used in the West to treat thalassaemia but it also has side effects.
The treatment, which has now received regulatory approval and which is being offered by the biotech company CRISPR Therapeutics, is aimed at curing a blood disorder known as β - Thalassaemia by correcting the genetic mutation responsible for the condition.
At present, they are used to treat diseases such as thalassaemia, in which patients accumulate surplus iron from repeated blood transfusions.
If you are undertaking any medical treatment for thalassaemia, kidney failure, cancer etc., the sum spent on these treatments can get tax exemption.
Intellia Therapeutics and Novartis recently announced a partnership to work on a similar approach, while Sangamo Therapeutics is looking to expand their Zinc Finger Nuclease treatment trials to β - Thalassaemia in collaboration with Bioverativ.
Blood tests can show whether you have a higher chance of inherited disorders such as sickle cell anaemia and thalassaemia, and whether you have infections like HIV, hepatitis B or syphilis.
Screening tests for sickle cell and thalassaemia will tell you for certain whether you're a carrier or have these conditions.
The screening test for sickle cell and thalassaemia should be offered as early as possible before 10 weeks of pregnancy.
NOTE: Anemia is not always caused by iron deficiency — lead poisoning and thalassaemia can also produce anemia.
Down's syndrome, haemophilia, fragile X syndrome, sickle - cell anaemia, thalassaemia and a plethora of other disorders are also down to genetic defects.
The team attempted to modify the gene responsible for β - thalassaemia, a potentially fatal blood disorder, using a gene - editing technique known as CRISPR / Cas9.
Currently we are developing non-invasive tests for other conditions caused by mutations in a single gene, including cystic fibrosis, sickle cell anemia, and beta - thalassaemia.
Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta - thalassaemia, another inherited blood disorder.
Thalassaemia is the world's most common genetic disease and is caused by mutations in one or both of the genes that code for haemoglobin.
David Weatherall, a thalassaemia expert at the University of Oxford, UK, told New Scientist: «In principle I'm greatly encouraged by this work.
But Lo's team found more of the healthy allele, suggesting that the fetus had inherited the correct maternal copy of the gene and would not develop beta - thalassaemia.
The woman's unborn child inherits one allele from its mother and one from its father, which gives it a 25 percent chance of getting two healthy alleles; a 50 percent chance of one faulty allele and one healthy; and a 25 percent chance of getting two mutated alleles that together would cause it to develop beta - thalassaemia, a potentially fatal blood disorder caused by low oxygen uptake.
Diana W. Bianchi, a reproductive geneticist at Tufts University School of Medicine in Boston, Mass., agrees that fetal genomes could inform treatment in the future, but she questions the need to replace conventional genetic testing with whole - genome sequencing to diagnose genetic diseases such as beta - thalassaemia and cystic fibrosis.
If they were present in equal proportions, it would mean that the child had indeed inherited the disease mutation from its mother, giving it a double dose of faulty genes that would lead to beta - thalassaemia.
Zbigniew Dominski and Ryszard Kole of the University of North Carolina's Lineberger Comprehensive Cancer Center in Chapel Hill, tested their method on genetic material from patients with b - thalassaemia, a severe type of anaemia.
The thalassaemia group of diseases, for instance, causes iron overload both pathologically and indirectly as a consequence of the massive blood transfusions used to treat the disease.
The work, led by Junjiu Huang, a gene - function researcher at Sun Yat - sen University in Guangzhou, involved attempts to modify the gene underlying the blood disorder β - thalassaemia.
The disease it targeted, beta - thalassaemia, can already be detected by pre-implantation embryonic screening during IVF.
Alpha - haemoglobin stabilising protein is a quantitative trait gene that modifies the phenotype of beta - thalassaemia.
Indeed, a lot of the fuss about the possibility of germline gene editing is misplaced, because there are very few instances where it would be necessary to correct a gene defect (which was the ultimate aim of the work reported here), because alternative techniques, notably pre-implantation genetic diagnosis (PGD), can be used to choose embryos for implantation that would not develop diseases such as beta - thalassaemia
Transfusion independence and HMGA2 activation after gene therapy of human β - thalassaemia.
Earlier this year, Chinese scientists caused an international furor when they reported that they had taken 86 human embryos and attempted to modify the gene that causes β - thalassaemia, a blood disorder that is potentially fatal.
Β - Thalassaemia is a blood condition that negatively impacts the production of haemoglobin, limiting the patient's ability to transport oxygen.
Patients in Europe are going to be offered a revolutionary gene - splicing therapy this year for the first time to treat the blood disorder beta - thalassaemia.
Expenses incurred by the individual on the treatment of critical ailments like Cancer, AIDS, neurological diseases, Parkinson's disease, Haemophilia, Thalassaemia and chronic renal failure, are tax deductible.
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