Help for patients with sickle cell disease may soon come from gene editing to fix the mutation that causes the disease and boost the patient's own protective fetal hemoglobin.
Jun 3, 2019
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Congenital heart disease occurs in up to 1% of live births, and the infants who are affected may require multiple surgeries, life-long medication, or heart transplants. In many patients, the exact cause of congenital heart ...
May 30, 2019
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A close look at the rapidly developing zebrafish embryo is helping neuroscientists better understand the potential underpinnings of brain disorders, including autism and schizophrenia.
May 23, 2019
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In a world first, researchers from The Australian National University (ANU) have shown previously ignored rare genetic mutations are a major cause of lupus.
May 17, 2019
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Investigators at the University of British Columbia (UBC)/Centre for Molecular Medicine & Therapeutics (CMMT) and BC Children's Hospital have examined more than 25 years of data to reveal new insights into predicting the ...
May 16, 2019
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High cholesterol is a risk factor for heart disease, but not all forms of it are the same. An underdiagnosed genetic condition called familial hypercholesterolemia can cause dangerously high levels of cholesterol at an early ...
May 15, 2019
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Johns Hopkins Medicine researchers recently found a key structural differences in channels that regulate how and when heart and skeletal muscles contract. Identifying the differences between the two, say researchers, provides ...
May 15, 2019
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Scientists have discovered how mutations in DNA can cause neurodegenerative disease. The discovery is an important step towards better treatment to slow the progression or delay onset in a range of incurable diseases such ...
May 13, 2019
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Congenital hepatic fibrosis (CHF) is a rare genetic disease that causes malformation and fibrosis (scarring) of the liver. Occurring in roughly one out of every 20,000 births, CHF can lead to an enlarged liver, impaired blood ...
May 8, 2019
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An international clinical trial has found that a new drug for Huntington disease is safe, and that treatment with the drug successfully lowers levels of the abnormal protein that causes the debilitating disease in patients.
May 6, 2019
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