A team of scientists at the National Institutes of Health (NIH) used gene editing to fix the mutation that causes a form of the rare Tay-Sachs disease in mice, sparking hope that the approach could ...
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...
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New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same ...
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