Drugs / Adeno-associated virus-CFTR vector
last change Jul 2003 re-read 3 minutes ago

Adeno-associated virus-CFTR vector

also known as AAV-CFTR · rAAV-CFTR vector · tgAAVCF

Gene therapy (AAV / viral vector) targets CFTR via restoration

Developed for
cystic fibrosis
Investigated by
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · University of Florida

Trials 1

19992000200120022003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT00004533 Aug 1999 → Aug 2002 cystic fibrosis National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Completed No outcome recorded

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as Adeno-associated virus-CFTR vector ClinicalTrials.gov intervention name — accepted as the source's own label NCT00004533 ↗
Known as AAV-CFTR “adeno-associated virus-CFTR (AAV-CFTR) vector” NCT00004533 ↗
Known as rAAV-CFTR vector “Doses of the rAAV-CFTR vector (tgAAVCF) ranging from 3 x 10(1) to 1 x 10(9) replication units (RU)” PMID 12885347 ↗ Jul 2003
Known as tgAAVCF “Doses of the rAAV-CFTR vector (tgAAVCF) ranging from 3 x 10(1) to 1 x 10(9) replication units (RU)” PMID 12885347 ↗ Jul 2003
Action Restore “stable correction of the CF defect in CF bronchial epithelial cells and stable expression of CFTR” PMID 12885347 ↗ Jul 2003
Modality Gene therapy (AAV / viral vector) “Recombinant adeno-associated serotype 2-based vectors (rAAV2)” PMID 12885347 ↗ Jul 2003
Target CFTR “rAAV2 vectors expressing the cystic fibrosis transmembrane conductance regulator (CFTR) gene” PMID 12885347 ↗ Jul 2003