>On the surface, cystic fibrosis and Tay-Sachs disease have nothing in common. Although both are inherited genetic disorders, one causes thick mucus buildup in the lungs, making it progressively harder to breathe; the other gradually leads to a buildup of fatty molecules that kills brain cells.
>But under the hood, the diseases share a common villain: Nonsense mutations.
>Like a molecular “stop sign,” these mutations instruct cells to abandon making certain proteins, resulting in truncated versions that don’t work and lead to disease. Gene-editing tools can correct mutated genes by targeting them one by one for each disease. While this approach can save lives, it takes time and a lot of resources to develop.
1 Comment
>On the surface, cystic fibrosis and Tay-Sachs disease have nothing in common. Although both are inherited genetic disorders, one causes thick mucus buildup in the lungs, making it progressively harder to breathe; the other gradually leads to a buildup of fatty molecules that kills brain cells.
>But under the hood, the diseases share a common villain: Nonsense mutations.
>Like a molecular “stop sign,” these mutations instruct cells to abandon making certain proteins, resulting in truncated versions that don’t work and lead to disease. Gene-editing tools can correct mutated genes by targeting them one by one for each disease. While this approach can save lives, it takes time and a lot of resources to develop.
>Why not aim for the common villain?