Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not...
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Method that silences specific genes while leaving others unaffected could aid research and therapy
Fusion of translational repressors to dPspCas13b enhances translational repression efficacy. Credit: Nature Communications (2024). DOI: 10.1038/s41467-024-46412-7 RIKEN researchers have developed...
