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CFTR

—CF Transmembrane Conductance Regulator

Chromosome: 7

Location: 7:117287120-117715971

OMIM: 602421

Ensembl: ENSG00000001626

Official·OMIM:602421 Official·ENSG00000001626

Encodes a chloride channel protein. Mutations cause cystic fibrosis.

Treatment Roadmap — CFTR

Preclinical

Phase 1

4 trial

Phase 2

18 trial

Phase 3

17 trial

Approved

Clinical trials have approximately 10% success rate. This roadmap shows the current status, not guaranteed outcomes.

Associated Diseases

Cystic Fibrosis

Autosomal RecessivePrevalence: ~100Krespiratory

Official·OMIM:219700 Official·ORPHA586

Summary

Clinical Trial

Approved Therapy

Registered Variant