Precision gene editing: From proof-of-concept to curative therapies.
Gene editing therapies are moving from rare diseases to a broader pipeline including blood disorders and immune deficiencies, though delivery barriers remain.
This review in Trends in Molecular Medicine synthesizes the rapidly evolving landscape of precision gene editing therapeutics, tracing the translational journey from initial CRISPR demonstrations to regulatory approvals for hemoglobinopathies and emerging pipeline for lysosomal storage disorders, immune deficiencies, and neurological conditions. The analysis highlights key remaining barriers including delivery, off-target effects, and health equity challenges that must be addressed to realize the curative potential of gene editing for rare disease patients globally.
What the study was
- Study design
- Review
- Category
- Drug Development
- Maturity
- Validated
- Journal
- Trends Mol Med
Why it surfaced
High-impact Cell Press review journal; comprehensive treatment of the gene editing curative therapy pipeline; directly relevant to rare disease monitoring watchlist; covers recently approved therapies and near-term pipeline.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.