Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a ...
Mar 6, 2024
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DNA, often thought of as "the blueprint of life," contains instructions for building proteins that cells need to survive and function properly. But DNA isn't perfect and errors can occur during replication. Sometimes, this ...
Feb 19, 2024
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There are over 7,000 different rare genetic diseases, and often it can be a significant challenge and take a long time to receive a correct diagnosis.
Jan 25, 2024
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Immunotherapies that target the CD20 antigen have revolutionized how patients with a variety of blood cancers and hematologic disorders have been treated. However, many patients develop resistance to these treatments due ...
Nov 16, 2023
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A UCLA-led team has identified an essential internal control mechanism that can promote the maturation of human stem cell-derived heart muscle cells, offering a deeper understanding of how heart muscle cells develop from ...
Oct 18, 2023
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A technique that enables scientists to record gene mutations and patterns of gene activity in individual cells has been extended to cover RNA splicing as well, in a study led by researchers at Weill Cornell Medicine, the ...
Aug 14, 2023
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So far, it has not been possible to explain the causes of around half of all rare hereditary diseases. A Munich research team has developed an algorithm that predicts the effects of genetic mutations on RNA formation six ...
Jun 1, 2023
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Pancreatic ductal adenocarcinoma (PDAC) is the most common form of pancreatic cancer. It's also one of the deadliest. More than 90% of PDAC patients die within five years of diagnosis. Usually, by the time the cancer is identified, ...
Apr 26, 2023
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Why do some people with a genetic mutation associated with leukemia remain healthy, while others with the same mutation develop the blood cancer? In a new study published in Blood, scientists from the USC Stem Cell laboratory ...
Mar 23, 2023
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Researchers at Lund University Faculty of Medicine have determined a novel mechanism linking the metabolism of ribonucleic acids, RNA, to the development of leukemia in myelodysplastic syndrome (MDS) patients. In a study ...
Mar 21, 2023
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