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Clinical Reviews in Allergy & Immunology

Erschienen in:

01.12.2008

Gene Therapy for Cystic Fibrosis

verfasst von: Christian Mueller, Terence R. Flotte

Erschienen in: Clinical Reviews in Allergy & Immunology | Ausgabe 3/2008

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Abstract

Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane conductance regulator (CFTR) gene that lead to defective ion transport in the conducting pulmonary airways and exocrine glands. Through a process that is not fully understood, CFTR defects predispose affected patients to chronic endobronchial infections with organisms such as Pseudomonas aeruginosa and Staphylococcus aureus. Following the discovery of the CFTR gene in 1989, CF became one of the primary targets for gene therapy research. Early enthusiasm surrounded the new field of gene therapy during most of the 1990s and it led academics and clinicians on a big effort to apply gene therapy for cystic fibrosis. Clinical studies have been pursued using recombinant adenovirus, recombinant adeno-associated virus, cationic liposomes, and cationic polymer vectors. Although to this date no dramatic therapeutic benefits have been observed, a lot of information has been gained from the pre-clinical and clinical studies that were performed. This learning curve has led to the optimization of vector technology and an appreciation of immune and mechanical barriers that have to be overcome for successful delivery.

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Titel: Gene Therapy for Cystic Fibrosis
verfasst von: Christian Mueller
Terence R. Flotte
Publikationsdatum: 01.12.2008
Verlag: Humana Press Inc
Erschienen in: Clinical Reviews in Allergy & Immunology / Ausgabe 3/2008
Print ISSN: 1080-0549
Elektronische ISSN: 1559-0267
DOI: https://doi.org/10.1007/s12016-008-8080-3

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Wer sich an einem Essensrest verschluckt und um Luft ringt, benötigt vor allem rasche Hilfe. Dass Umstehende nur in jedem zweiten Erstickungsnotfall bereit waren, diese zu leisten, ist das ernüchternde Ergebnis einer Beobachtungsstudie aus Japan. Doch es gibt auch eine gute Nachricht.

Real-World-Daten sprechen eher für Dupilumab als für Op.

14.05.2024 Rhinosinusitis Nachrichten

Zur Behandlung schwerer Formen der chronischen Rhinosinusitis mit Nasenpolypen (CRSwNP) stehen seit Kurzem verschiedene Behandlungsmethoden zur Verfügung, darunter Biologika, wie Dupilumab, und die endoskopische Sinuschirurgie (ESS). Beim Vergleich der beiden Therapieoptionen war Dupilumab leicht im Vorteil.

Schwindelursache: Massagepistole lässt Otholiten tanzen

14.05.2024 Benigner Lagerungsschwindel Nachrichten

Wenn jüngere Menschen über ständig rezidivierenden Lagerungsschwindel klagen, könnte eine Massagepistole der Auslöser sein. In JAMA Otolaryngology warnt ein Team vor der Anwendung hochpotenter Geräte im Bereich des Nackens.

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Die Highlights vom Kongress des American College of Cardiology 2024

Springer Medizin

Gene Therapy for Cystic Fibrosis

Abstract

Neu im Fachgebiet HNO

Betalaktam-Allergie: praxisnahes Vorgehen beim Delabeling

Eingreifen von Umstehenden rettet vor Erstickungstod

Real-World-Daten sprechen eher für Dupilumab als für Op.

Schwindelursache: Massagepistole lässt Otholiten tanzen

Update HNO

Die Highlights vom Kongress des American College of Cardiology 2024

Springer Medizin

Abstract

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Weitere Artikel der Ausgabe 3/2008

Overview of Lung Transplantation

Newborn Screening for Cystic Fibrosis

Diagnosis of Cystic Fibrosis

Cystic Fibrosis: Evolution from a Fatal Disease of Infancy with a Clear Phenotype to a Chronic Disease of Adulthood with Diverse Manifestations

Atypical Cystic Fibrosis and CFTR-Related Diseases

P. aeruginosa Biofilms in CF Infection

Neu im Fachgebiet HNO

Betalaktam-Allergie: praxisnahes Vorgehen beim Delabeling

Eingreifen von Umstehenden rettet vor Erstickungstod

Real-World-Daten sprechen eher für Dupilumab als für Op.

Schwindelursache: Massagepistole lässt Otholiten tanzen

Update HNO