Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues. One technology platform ...
May 18, 2011
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A razor nick in the shower? A fall on a slippery floor? The average person simply calls it bad karma, but for people with hemophilia such episodes can cause prolonged blood loss.
Jun 27, 2011
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Children with hemophilia A require three to four infusions each week to prevent bleeding episodes, chronic pain and joint damage. The effect on quality of life can be significant, due to time and discomfort associated with ...
Apr 24, 2015
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Growing up in Devon in the 1980s, brothers James and Matt (names have been changed) had to be careful with their antics – a cut or bruise could land them in hospital.
Jan 15, 2019
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Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end. Their bodies lack the ability to make the clotting ...
Jul 24, 2015
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A single gene therapy injection could dramatically reduce the bleeding risk faced by people with hemophilia B, finds a study involving UCL researchers.
Jul 20, 2022
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Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn't broken, a fall or a simple toe stub can become a serious medical issue: internal ...
Mar 10, 2020
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(Medical Xpress) -- Scientists have used a gene therapy tool that acts like intelligent molecular scissors to correct the key gene defect in mice with hemophilia B, a disease that can lead to uncontrolled bleeding. The intervention ...
Jun 28, 2011
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A St. Jude Children's Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene ...
Jun 4, 2021
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In fact, the studies estimate that only 4 percent of the powerful drug's use in U.S. hospitals from 2000 through 2008 was for treating hemophilia patients, while an enormous 96 percent involved cases of heart surgery, trauma, ...
Apr 18, 2011
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