Consider for a moment that your life is dominated by spontaneous nosebleeds, chronic stomach hemorrhaging, persistent anemia and a complex constellation of other manifestations, some potentially life-threatening.
Researchers from multiple institutions in China have found a way to use gene editing to reactivate dormant fetal oxygen-transporting proteins in adult blood cells to potentially reverse a wide range of blood disorders.
Having a rare genetic disease is actually pretty common. Rare diseases affect approximately 1 in 10 individuals, and more than 30 million people in the U.S. have a rare disease diagnosis. What makes them rare is that these ...
A team of sister scientists is on a mission to find a cure for one of the most common cancers, uterine corpus endometrial cancer (UCEC). UCEC, the fourth deadliest cancer in women, is often not detected until it is already ...
Mar 14, 2023
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Rett syndrome, a rare genetic disorder that causes mental retardation, autism, and physical deformities, has no cure. However, a small clinical trial has found that a growth factor known as IGF1 can help treat some symptoms ...
Jun 23, 2014
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For many inherited diseases, such as cystic fibrosis or Huntington disease, the disease-causing genetic mutation damages or removes a protein that has an essential role in the body. This protein defect is the root cause of ...
Aug 8, 2013
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Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, ...
Apr 22, 2024
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A new lab assay developed by researchers at Fred Hutch Cancer Center could make diagnosis and treatment of small-cell lung cancer and non-small cell lung cancer easier. The work is published in the journal Science Advances.
Apr 10, 2024
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A single genetic test could potentially replace the current two-step approach to diagnosing rare developmental disorders in children. This shift could enable earlier diagnoses for families and save the NHS vital resources.
Mar 28, 2024
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A U.K. trial has found a DNA-tailored diet could help manage blood glucose and reduce risk of progressing to type 2 diabetes in high-risk individuals.
Mar 7, 2024
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