Lessons learned from lung and liver in-vivo gene therapy: implications for the future

A guide to manufacturing CAR T cell therapies

(2018) Philipp Vormittag et al.   CURRENT OPINION IN BIOTECHNOLOGY

Developmental mechanisms and adult stem cells for therapeutic lung regeneration

(2018) Joo-Hyeon Lee et al.   DEVELOPMENTAL BIOLOGY

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9–Like Vectors: Putting Patients First

(2018) Terence R. Flotte et al.   HUMAN GENE THERAPY

Role of Basal Cells in Producing Persistent Lentivirus-Mediated Airway Gene Expression

(2018) Nigel Farrow et al.   HUMAN GENE THERAPY

Transducing Airway Basal Cells with a Helper-Dependent Adenoviral Vector for Lung Gene Therapy

(2018) Huibi Cao et al.   HUMAN GENE THERAPY

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

(2018) Nicole K. Paulk et al.   MOLECULAR THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Anticipating the clinical adoption of regenerative medicine: building institutional readiness in the UK

(2018) John Gardner et al.   Regenerative Medicine

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

A single-cell atlas of the airway epithelium reveals the CFTR-rich pulmonary ionocyte

(2018) Lindsey W. Plasschaert et al.   NATURE

A revised airway epithelial hierarchy includes CFTR-expressing ionocytes

(2018) Daniel T. Montoro et al.   NATURE

Featured Article: Electroporation-mediated gene delivery of surfactant protein B (SP-B) restores expression and improves survival in mouse model of SP-B deficiency

(2017) Rebecca C Barnett et al.   EXPERIMENTAL BIOLOGY AND MEDICINE

Optimization of lentiviral vector production for scale-up in fixed-bed bioreactor

(2017) A J Valkama et al.   GENE THERAPY

Novel GP64 envelope variants for improved delivery to human airway epithelial cells

(2017) P L Sinn et al.   GENE THERAPY

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

(2017) Dolan Sondhi et al.   HUMAN GENE THERAPY

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

(2017) Christian Mueller et al.   MOLECULAR THERAPY

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

(2017) Florie Borel et al.   MOLECULAR THERAPY

Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome

(2017) Grant J Logan et al.   NATURE GENETICS

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Enhancing titres of therapeutic viral vectors using the transgene repression in vector production (TRiP) system

(2017) H. E. Maunder et al.   Nature Communications

Pseudotyped Lentiviral Vectors: One Vector, Many Guises

(2017) Alok V. Joglekar et al.   Human Gene Therapy Methods

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Delivery of ENaC siRNA to epithelial cells mediated by a targeted nanocomplex: a therapeutic strategy for cystic fibrosis

(2017) Maria D. I. Manunta et al.   Scientific Reports

Challenges of up-scaling lentivirus production and processing

(2016) Alexandra McCarron et al.   JOURNAL OF BIOTECHNOLOGY

Barriers to inhaled gene therapy of obstructive lung diseases: A review

(2016) Namho Kim et al.   JOURNAL OF CONTROLLED RELEASE

77. Antigen-Specific Modulation of Capsid Immunogenicity with Tolerogenic Nanoparticles Results in Successful AAV Vector Readministration

(2016) Amine Meliani et al.   MOLECULAR THERAPY

Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients

(2016) Irene Gil-Farina et al.   MOLECULAR THERAPY

A universal system to select gene-modified hepatocytes in vivo

(2016) Sean Nygaard et al.   Science Translational Medicine

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

(2016) Eric W F W Alton et al.   THORAX

Liver-targeted hydrodynamic gene therapy: Recent advances in the technique

(2016) Takeshi Yokoo et al.   WORLD JOURNAL OF GASTROENTEROLOGY

Multilineage transduction of resident lung cells in vivo by AAV2/8 for α1-antitrypsin gene therapy

(2016) Julia G Payne et al.   Molecular Therapy-Methods & Clinical Development

The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

(2016) Sirkka Kyostio-Moore et al.   Molecular Therapy-Methods & Clinical Development

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

(2016) Nathalie Clément et al.   Molecular Therapy-Methods & Clinical Development

Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1

(2015) R Castello et al.   GENE THERAPY

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

(2015) Kenneth I. Berns et al.   HUMAN GENE THERAPY

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

(2015) Lili Wang et al.   MOLECULAR THERAPY

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

(2015) Jean-Charles Nault et al.   NATURE GENETICS

Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy

(2015) Panagiotis Mastorakos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Aerosolized Medications for Gene and Peptide Therapy

(2015) B. L. Laube   Respiratory Care

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

(2015) Alessio Cantore et al.   Science Translational Medicine

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

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

(2014) N. Nair et al.   BLOOD

Characterization of Pulmonary T Cell Response to Helper-Dependent Adenoviral Vectors following Intranasal Delivery

(2014) R. Kushwah et al.   JOURNAL OF IMMUNOLOGY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Intramuscular Injection of AAV8 in Mice and Macaques Is Associated with Substantial Hepatic Targeting and Transgene Expression

(2014) Jenny A. Greig et al.   PLoS One

Aerosol Delivery of DNA/Liposomes to the Lung for Cystic Fibrosis Gene Therapy

(2014) Lee A. Davies et al.   Human Gene Therapy Clinical Development

Phase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin Deficiency

(2014) Maria J. Chiuchiolo et al.   Human Gene Therapy Clinical Development

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

(2013) David M. Markusic et al.   EMBO Molecular Medicine

Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

(2013) Andrea Annoni et al.   EMBO Molecular Medicine

Gene therapy for PIDs: Progress, pitfalls and prospects

(2013) Sayandip Mukherjee et al.   GENE

OneBac: Platform for Scalable and High-Titer Production of Adeno-Associated Virus Serotype 1–12 Vectors for Gene Therapy

(2013) Mario Mietzsch et al.   HUMAN GENE THERAPY

A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro

(2013) Jiamiao Lu et al.   MOLECULAR THERAPY

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Efficient Biodistribution and Gene Silencing in the Lung epithelium via Intravenous Liposomal Delivery of siRNA

(2013) Jana McCaskill et al.   Molecular Therapy-Nucleic Acids

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

Positive regulation of hepatic miR-122 expression by HNF4α

(2011) Zhen-Ya Li et al.   JOURNAL OF HEPATOLOGY

A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A

(2011) Hideto Matsui et al.   MOLECULAR THERAPY

Preparing for the First Breath: Genetic and Cellular Mechanisms in Lung Development

(2010) Edward E. Morrisey et al.   DEVELOPMENTAL CELL

The SP-C promoter facilitates alveolar type II epithelial cell-specific plasmid nuclear import and gene expression

(2010) J V DeGiulio et al.   GENE THERAPY

Lung deposition of inhaled  1-proteinase inhibitor in cystic fibrosis and  1-antitrypsin deficiency

(2009) P. Brand et al.   EUROPEAN RESPIRATORY JOURNAL

Progress and Prospects: prospects of repeated pulmonary administration of viral vectors

(2009) P L Sinn et al.   GENE THERAPY

Induction of Immune Tolerance to FIX Following Muscular AAV Gene Transfer Is AAV-dose/FIX-level Dependent

(2009) Meagan E Kelly et al.   MOLECULAR THERAPY

Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium

(2009) Wuping Li et al.   MOLECULAR THERAPY

Directed evolution of adeno-associated virus to an infectious respiratory virus

(2009) K. J. D. A. Excoffon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Detection of expression of influenza virus receptors in tissues of BALB/c mice by histochemistry

(2009) Zhang-Yong Ning et al.   VETERINARY RESEARCH COMMUNICATIONS

Ciliated epithelial cell lifespan in the mouse trachea and lung

(2008) Emma L. Rawlins et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Enhanced Lung Gene Expression After Aerosol Delivery of Concentrated pDNA/PEI Complexes

(2008) Lee A Davies et al.   MOLECULAR THERAPY

Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro

(2008) Maria P Limberis et al.   MOLECULAR THERAPY

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

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY