- Adeno-associated virus vectors in clinical trials
Barrie J Carter
Targeted Genetics, Seattle, WA 98101, USA
Hum Gene Ther 16:541-50. 2005
- A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease
M L Aitken
Department of Medicine and Pediatrics, University of Washington, Seattle, 98195, USA
Hum Gene Ther 12:1907-16. 2001
..RNA-specific PCR did not detect vector-derived mRNA. This Phase I trial shows that aerosolized tgAAVCF is safe and widely delivered to the proximal airways of CF subjects by nebulization...
- Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge
Institute of Ophthalmology, University College London, London, United Kingdom
N Engl J Med 358:2231-9. 2008
..These findings provide support for further clinical studies of this experimental approach in other patients with mutant RPE65. (ClinicalTrials.gov number, NCT00643747 [ClinicalTrials.gov].)...
- AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice
Robin J Ziegler
Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701 9322, USA
Mol Ther 9:231-40. 2004
..Together, these results demonstrate that AAV2-mediated gene transfer that limits the expression of alpha-galactosidase A to the liver may be a viable strategy for treating Fabry disease...
- A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
John A Wagner
Department of Molecular Pharmacology, Stanford CCSR Building, Stanford, CA 94305 5174, USA
Hum Gene Ther 13:1349-59. 2002
..In summary, this Phase II trial confirms the safety of tgAAVCF but provides little support of its efficacy in the within-patient controlled sinus study. Various potentially confounding factors are discussed...
- Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi
Jodi L McBride
Departments of Internal Medicine, Molecular Physiology and Biophysics, and Neurology, University of Iowa, Iowa City, IA 52242, USA
Proc Natl Acad Sci U S A 105:5868-73. 2008
- Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial
Richard B Moss
Department of Pediatrics, Stanford University, Stanford, CA 94305, and Division of Respiratory Diseases, Children s Hospital Boston, MA 02115, USA
Hum Gene Ther 18:726-32. 2007
..Because gene transfer is the simplest, most basic way to correct the underlying genetic defect that leads to disease in CF, further research is warranted to develop an effective gene transfer agent for the treatment of CF...
- Workshop on long-term follow-up of participants in human gene transfer research
Minneapolis, MN, USA
Mol Ther 10:976-80. 2004
- Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy
Terence R Flotte
Department of Pediatrics, University of Florida, Gainesville, 32610, USA
Hum Gene Ther 16:921-8. 2005
..The observation of sizeable physiological correction in the face of low mRNA levels may reflect the regulatory role of low levels of CFTR protein as an activator of other chloride channels...
- Adeno-associated virus and the development of adeno-associated virus vectors: a historical perspective
Barrie J Carter
Targeted Genetics Corporation, Seattle, WA 98101, USA
Mol Ther 10:981-9. 2004
- Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study
Terence R Flotte
Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610 0296, USA
Hum Gene Ther 14:1079-88. 2003
..These data indicate the need for continued evaluation of rAAV-CFTR vectors in additional clinical trials...
- Nonclinical toxicology in support of licensure of gene therapies. Arlington, VA, USA, March 13-14, 2003
Robert M Frederickson
TherImmune Research Corporation, Gaithersburg, MD 20879, USA
Mol Ther 8:8-10. 2003