Researchers from MIT, the McGovern Institute for Brain Research at MIT, the Howard Hughes Medical Institute, and the Broad Institute of MIT and Harvard have developed a new way to deliver molecular therapies to cells. The ...
Aug 19, 2021
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Scientists have, for the first time, corrected a disease-causing mutation in early stage human embryos with gene editing. The technique, which uses the CRISPR-Cas9 system, corrected the mutation for a heart condition at the ...
Aug 2, 2017
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Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy ...
Dec 31, 2015
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A mutation in a protein regulating natural killer (NK) cells' function is at the root of immune deficiency in some people with a rare genetic condition characterized by cognitive and developmental delay, seizures, and other ...
Apr 9, 2024
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New research from Oregon Health & Science University for the first time reveals the function of a little-understood junction between cells in the brain that could have important treatment implications for conditions ranging ...
Jan 12, 2024
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Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals' genetic ancestry. PKU causes an amino acid—called phenylalanine (Phe)—to build ...
Nov 2, 2023
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Researchers have used CRISPR gene editing, stem cells and human neurons to study the impact of a gene that is commonly mutated in autism. This new study, published today in The American Journal of Human Genetics, ties mutations ...
Oct 5, 2023
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Genetic alterations that give rise to a rare, fatal disorder known as MOGS-CDG paradoxically also protect cells against infection by viruses. Now, scientists at the Lewis Katz School of Medicine at Temple University have ...
May 19, 2023
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A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does ...
May 4, 2023
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University of Minnesota researchers and their collaborators at the National Cancer Institute (NCI) investigated the role of a new intracellular checkpoint gene in regulating T-cell function against solid tumors. Known as ...
Sep 13, 2022
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