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Tag: geneediting

Gene-editing enzymes imaged in 3D

Gene-editing enzymes imaged in 3D

Science
July 8 (UPI) -- Scientists have for the first time captured high-definition, 3D images of enzymes in the process of cutting DNA strands. The breakthrough -- described Monday in the journal Nature Structural and Molecular Biology -- helped scientists see exactly how the gene-editing technology called CRISPR-Cas9 works, and could, in the future, help researchers design a more efficient and precise version of the technology. The findings could also help scientists better understand -- and eventually, treat and prevent -- diseases caused by DNA mutations, including cancer, sickle cell anemia, Tay-Sachs disease, Huntington's disease and many others. "It is exciting to be able to see at such a high level of detail how Cas9 actually works to cut and edit DNA strands," lead study author Sriram S...
Gene-editing hope for muscular dystrophy

Gene-editing hope for muscular dystrophy

Science
Scientists have for the first time used gene-editing to treat Duchenne muscular dystrophy in a large mammal, a significant step towards effective treatment for people with the disorder.The condition, which has no cure, leads to loss of muscle function and strength and ultimately an early death.But in a study on dogs, scientists were able to partially restore the key protein people with DMD cannot make.They hope in the future to test the technique in people. Green light for muscular dystrophy drug First gene-editing in human body attempt Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease in children and almost entirely affects boys and young men - about 2,500 of them in the UK have the condition. Children born with the degenerative ...
Gene-editing patient applications closer with new technique

Gene-editing patient applications closer with new technique

Health
April 13 (UPI) -- A new technique in gene editing greatly improves the technology's precision, so much so that it may one day treat some genetic diseases, according to a new study.At the University of Alberta, researchers have improved the accuracy using the guided biomachine in the body to seek out defective gene sequences in each cell and edit in the correct information. Their findings were published Friday in Nature Communications.They have filed a patent on their discovery and want to partner with a pharmaceutical company to use it for treatment of diseases, including muscular dystrophy, hemophilia and some cancers.The tool called Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR, combines a scissor-like protein called Cas9 and a guide molecule that seeks a precise s...
First gene-editing in human body attempt

First gene-editing in human body attempt

Health
Gene-editing has been attempted on cells inside a patient, in a world first by doctors in California.Brian Madeux, 44 from Arizona, was given the experimental treatment to try to correct a defect in his DNA that causes Hunter's syndrome. Mr Madeux says he was prepared to take part in the trial as he is "in pain every second of the day".It is too soon to know whether or not the gene-editing has worked in Mr Madeux's case. Hunter's syndrome is rare. Patients are born without the genetic instructions for an enzyme that breaks down long sugary molecules called mucopolysaccharides.Instead, they build up in the body and damage the brain and other organs. Severe cases are often fatal. "I actually thought I wouldn't live past my early 20s," said Mr Madeux.Patients need regular enzyme replacement t...