Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

(2015) Eric W F W Alton et al.   Lancet Respiratory Medicine

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs

(2015) Timothy C. Nichols et al.   Human Gene Therapy Clinical Development

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

(2014) A. Lattanzi et al.   HUMAN MOLECULAR GENETICS

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

(2014) Stéphane Palfi et al.   LANCET

Gene therapy of primary T cell immunodeficiencies

(2013) Alain Fischer et al.   GENE

Current progress on gene therapy for primary immunodeficiencies

(2013) L Zhang et al.   GENE THERAPY

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy

(2012) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Lentiviral Vector Gene Transfer to Porcine Airways

(2012) Patrick L Sinn et al.   Molecular Therapy-Nucleic Acids

Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues

(2011) Cynthia C Bartholomae et al.   MOLECULAR THERAPY

Validation of recombinant Sendai virus in a non-natural host model

(2010) U Griesenbach et al.   GENE THERAPY

Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes

(2010) Katsuyuki Mitomo et al.   MOLECULAR THERAPY

A Ubiquitous Chromatin Opening Element (UCOE) Confers Resistance to DNA Methylation–mediated Silencing of Lentiviral Vectors

(2010) Fang Zhang et al.   MOLECULAR THERAPY

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

Ion transport across CF and normal murine olfactory and ciliated epithelium

(2009) B. R. Grubb et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer

(2009) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Development of inducible EIAV-based lentiviral vector packaging and producer cell lines

(2009) H J Stewart et al.   GENE THERAPY

Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages

(2009) Andrew A. Wilson et al.   JOURNAL OF CLINICAL INVESTIGATION

Validation of Nasal Potential Difference Measurements in Gut-Corrected CF Knockout Mice

(2008) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Lentivirus Vector Can Be Readministered to Nasal Epithelia without Blocking Immune Responses

(2008) P. L. Sinn et al.   JOURNAL OF VIROLOGY

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY