Advances in Gene Editing Offer New Hope for Rare Diseases

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Gene‑editing technologies are entering a new era, with researchers developing treatments that target the root causes of rare genetic disorders. Instead of managing symptoms, these therapies aim to correct faulty genes directly.

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Recent breakthroughs include successful trials for conditions once considered untreatable. While challenges remain — including cost and long‑term safety — the progress is reshaping expectations for genetic medicine.

For families affected by rare diseases, gene editing represents a future filled with possibilities.