geoscience.net logo
+ Resolve Article
+ Follow Us
Follow on FacebookFollow on Facebook
Follow on TwitterFollow on Twitter

+ Translate
+ Subscribe to Site Feed
GeoScience Most Shared ContentMost Shared Content

Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy


, : Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Current Opinion in Molecular Therapeutics 3(5): 497-502

Cystic fibrosis (CF) is an autosomal recessive inherited disorder that affects approximately 30,000 North Americans. Defects in the CF transmembrane conductance regulator (CFTR) gene lead to altered secretions from exocrine glands and the pulmonary airways, to a heightened susceptibility to airway infections with Pseudomonas aeruginosa, and to severe airway inflammation. Early attempts to develop a genetic therapy for CF have not met with great clinical success, but these efforts have driven the development of viral gene transfer technology for in vivo gene delivery. The recombinant adeno-associated virus (rAAV) system has proven to be safe for in vivo gene delivery in the airways of experimental animals and CF patients, although potential barriers to delivery have been identified. These barriers may limit the transduction efficiency of this vector, especially in the context of the inflamed airways of adolescent and adult CF patients. We anticipate that the use of alternative rAAV serotype capsids and other vector alterations, along with targeting the lungs of CF patients in the earlier stages of their disease, might eventually allow for these potential limitations to be overcome.

US$29.90

PMID: 11699895


Other references

das Neves, M.M., 1994: Alcoholism and pancreatic dysfunction. Revista Da Associacao Medica Brasileira 40(1): 69-70

Ellis, J.P.; Camper, N.D., 1995: In vitro cultured cocklebur (Xanthium strumarium L.) responses to dimercaptopropanesulfonic acid and monosodium methanearsonate. Monosodium methanearsonate (MSMA) is an effective herbicide used for the control of Xanthium strumarium (cocklebur), a serious weed problem in cotton production. Recently, MSMA-resistant cocklebur was reported, which reduced MSMA effectiveness in...

Hutcheon, D.E.; Gold, H., 1972: Harry Gold, M.D., F.C.P., 1889-1972. Journal of Clinical Pharmacology and New Drugs 12(8): 303-305

Machicoane, G.; Felice, H.; Fogleman, J.; Hafalia, R.; Morgan, G.; Pan, H.; Prestemon, S.; Pozdeyev, E.; Rao, X.; Ren, H.; Tobos, L., 2016: Status of ECR ion sources for the Facility for Rare Isotope Beams (FRIB) (invited). Ahead of the commissioning schedule, installation of the first Electron Cyclotron Resonance (ECR) ion source in the front end area of the Facility for Rare Isotope Beam (FRIB) is planned for the end of 2015. Operating at 14 GHz, this first ECR wil...

Noguchi R.; Miyanaga N.; Kanoh S.; Koiso K., 1991: Intra arterial infusion chemotherapy for bladder cancer by insertion of catheter from inferior gluteal artery. Journal of Urology 145(4 SUPPL): 332A

Dambrauskiene,E.; Viskelis,P.; Karkleliene,R., 2008: Productivity and biochemical composition of Mentha piperita L of different origin. Biochemical composition analysis of peppermint of different origin was carried out at the Lithuanian Institute of Horticulture. <i>Mentha</i> x <i>piperita</i> L. collection samples from Lithuania and the cultivar 'Kra...

Willardson, J.M.; Bressel, E., 2004: Predicting a 10 repetition maximum for the free weight parallel squat using the 45 degrees angled leg press. The purpose of this research was to devise prediction equations whereby a 10 repetition maximum (10RM) for the free weight parallel squat could be predicted using the following predictor variables: 10RM for the 45 degrees angled leg press, body ma...

Lopes, C.Andréa.Facury.; Lerário, Aônio.Carlos.; Mion, Décio.; Koch, V.; Wajchenberg, B.Leo.; Rosenbloom, A.L., 2004: Ambulatory blood pressure monitoring (ABPM) in normotensive adolescents with type 1 diabetes. To evaluate 24-h ambulatory blood pressure monitoring (ABPM) for early detection of hypertension in adolescents with type 1 diabetes mellitus (DM1). Patients underwent fundoscopy, albuminuria determinations, two repeated autonomic cardiovascular t...

Karp, A.; Meza, L.C.; Leon, S.C. de, 1999: Water as the source of community empowerment: part 2. The paper shows how communities, when properly organized and trained, may be able to operate and maintain a water system well beyond its 'design life'. It follows up the developments in two adjacent communities in Guatemala, Panimaquip a...

Fraser, I.S., 2013: Added health benefits of the levonorgestrel contraceptive intrauterine system and other hormonal contraceptive delivery systems. It has been recognized for well over half a century that hormonal preparations designed as contraceptives are also capable of offering health benefits through the treatment and prevention of benign gynecological disease and even some systemic cond...