Modulating the activity of a kinase in motor neurons may help mitigate mitochondrial defects and other symptoms of spinal muscular atrophy, offering a new therapeutic avenue for the devastating disease, according to a Northwestern ...
Dec 5, 2023
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Medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions such as sickle cell disease and the findings of this study, published in Nature Medicine, offer important new ...
Nov 16, 2023
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Gene therapy has great promise for treating genetic diseases, and even for more common diseases such as atherosclerosis (hardening of the arteries). Over the past decade, the gene-editing technology CRISPR (Clustered Regularly ...
Nov 16, 2023
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Neuroscientists at the Faculty of Medicine and Surgery of the Catholic University, Rome, and the Fondazione Policlinico Universitario Agostino Gemelli IRCCS have genetically modified a molecule, the protein LIMK1, which is ...
Nov 15, 2023
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CAR T cell therapy, a powerful type of immunotherapy, has begun to revolutionize cancer treatment. Pioneered at Memorial Sloan Kettering Cancer Center (MSK), the therapy involves engineering a patient's T cells so they recognize ...
Nov 14, 2023
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A team of surgeons in New York has performed the world's first transplant of an entire eye in a procedure widely hailed as a medical breakthrough, although it isn't yet known whether the man will ever see through the donated ...
Nov 9, 2023
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Researchers at Duke University have adapted CRISPR technologies for high-throughput screening of gene function in human immune cells and discovered that a single master regulator of the genome can be used to reprogram a network ...
Nov 9, 2023
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Gene therapy adeno-associated viruses (AAVs)—viruses that can be engineered to deliver DNA to target cells—are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to long-term efficacy, ...
Nov 6, 2023
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Cancer cells are often a mess of mutations. About 20% to 25% of cancers involve mutations in a complex of molecules called SWI/SNF. Yet drugs designed to block SWI/SNF activity haven't always worked as expected. Researchers ...
Nov 3, 2023
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Researchers from the Shenzhen Institute of Advanced Technology (SIAT) of the Chinese Academy of Sciences (CAS) and their collaborators have developed a gene therapy strategy to selectively manipulate Parkinson's disease-affected ...
Nov 2, 2023
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