A new gene-editing technique called PERT offers a potential single-tool solution for numerous genetic disorders caused by nonsense mutations. This method, described in Nature, instructs cells to read ...
An experimental drug suggests that a 'path is clearly achievable' to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic ...
Premature termination codons (PTCs) account for 10 to 20% of genetic diseases in humans. The gene inactivation resulting from PTCs can be counteracted by the use of drugs stimulating PTC readthrough, ...
BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced an award to Eloxx Pharmaceuticals Inc. for up to $15.9 million in additional funding to support the expansion of its ...
Although previously thought to be neutral, research in yeast suggests that synonymous point mutations may have strongly nonneutral effects. A group of researchers from the University of Michigan (MI, ...
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Breakthrough for genetic diseases: New tool can fix ‘nonsense’ mutations, including haemoglobin defects
A new gene-editing technique known as PERT has shown the ability to correct a wide range of disorders caused by “nonsense” mutations, including forms that interfere with haemoglobin production. The ...
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