Scientists halt genetic disease progression using custom DNA blockers

Researchers have demonstrated that synthetic DNA and protein molecules can prevent the repetitive genetic mutations that cause Friedreich's ataxia, a devastating inherited neurological disorder. The finding, published in Nucleic Acids Research, opens a potential treatment pathway for a rare disease with no cure—and validates a molecular strategy that could apply to other repeat-expansion disorders affecting thousands of patients.

Originaltitel: Large-scale expansions of Friedreich's ataxia GAA•TTC repeats in an experimental human system: role of DNA replication and prevention by LNA-DNA oligonucleotides and PNA oligomers