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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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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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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Researchers at Great Ormond Street Hospital for Children (GOSH) and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have used CRISPR/Cas9 technology to engineer donor T cells to try to treat seriously ill ...
Oct 27, 2022
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A novel gene editing technique developed by researchers at The Ohio State University Wexner Medical Center and College of Medicine is showing promise in correcting genetic mutations in Duchenne muscular dystrophy (DMD) and ...
Jun 25, 2021
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A gene that cured a man of HIV a decade ago has been successfully added to developing monkey embryos in an effort to study more potential treatments for the disease.
Nov 17, 2020
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When Wei Hsi (Ariel) Yeh was a young undergraduate student, one of her close friends went from normal hearing to complete deafness in the span of one month. He was 29 years old. No one knew why he lost his hearing; doctors ...
Jun 3, 2020
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For the first time scientists have identified how to halt kidney disease in a life-limiting genetic condition, which may pave the way for personalised treatment in the future.
Nov 16, 2018
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