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Scientists lay foundation for potential gene-editing therapy for late-onset Tay-Sachs disease
Scientists at the National Institutes of Health (NIH) have successfully reduced the severity of late-onset Tay-Sachs (LOTS) disease in human cell cultures and a mouse model by using a novel ...
Stewart Altman, wheelchair-bound, fights for cure as gene therapy shows promise. May 13, 2011— -- For 26 years, doctors could not piece together the medical puzzle of Stewart Altman's symptoms -- ...
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 ...
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