Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis.

Gene and cell-based therapies are considered to be potentially powerful new approaches for the management of cystic fibrosis (CF) lung disease. Despite tremendous efforts that have been made, especially in studies to understand the obstacles to gene delivery, major challenges to the application of these approaches remain to be solved. This article will review the advancements made and challenges remaining in the development of viral vector-mediated and cell-based approaches to treat patients with CF.

• Publication year

  2007

• Venue

  Molecular Therapy

• Publication date

  2007-02-01

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1038/SJ.MT.6300002PMID 17235299
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• Major challenges remain for applying these approaches, with obstacles to gene delivery singled out as a central limitation.

  Confidence 0.93

  박진우 (dztg5apj7m) extraction뀨 (7c402c1b98) reviewq (76h6bfydm6) review
• Viral vector-mediated and cell-based approaches are presented as promising new strategies for managing cystic fibrosis lung disease.

  Confidence 0.91

  박진우 (dztg5apj7m) extraction뀨 (7c402c1b98) reviewq (76h6bfydm6) review

• cell-based therapy

  therapy approach

  A treatment approach that uses cells as the therapeutic agent, including transplanted or engineered cells.

  Aliases: cell-based approach

  박진우 (dztg5apj7m) extraction뀨 (7c402c1b98) reviewq (76h6bfydm6) review
• cystic fibrosis lung disease

  disease

  The pulmonary disease manifestation of cystic fibrosis that is the target of the therapies discussed.

  Aliases: CF lung disease

  박진우 (dztg5apj7m) extraction뀨 (7c402c1b98) reviewq (76h6bfydm6) review
• gene delivery obstacles

  barrier

  Technical barriers that reduce the efficiency or practicality of delivering genetic material to airway cells.

  Aliases: obstacles to gene delivery, gene-delivery barriers

  박진우 (dztg5apj7m) extraction뀨 (7c402c1b98) reviewq (76h6bfydm6) review
• viral vector-mediated therapy

  therapy approach

  A gene-delivery strategy that uses viral vectors to introduce therapeutic material into target cells.

  Aliases: viral vector-mediated approach, viral vector therapy

  박진우 (dztg5apj7m) extraction뀨 (7c402c1b98) reviewq (76h6bfydm6) review

• Identification of the Cystic Fibrosis Gene : Chromosome Walking and Jumping Author ( s ) :

  2008cited by this paper
• Pluripotency of mesenchymal stem cells derived from adult marrow

  2007cited by this paper
• Assessment of cystic fibrosis transmembrane conductance regulator (CFTR) activity in CFTR-null mice after bone marrow transplantation.

  2006cited by this paper
• Limited restoration of cystic fibrosis lung epithelium in vivo with adult bone marrow-derived cells.

  2006cited by this paper
• Human Immunodeficiency Virus Type 1 Incorporated with Fusion Proteins Consisting of Integrase and the Designed Polydactyl Zinc Finger Protein E2C Can Bias Integration of Viral DNA into a Predetermined Chromosomal Region in Human Cells

  2006cited by this paper
• Influenza M2 envelope protein augments avian influenza hemagglutinin pseudotyping of lentiviral vectors

  2006cited by this paper
• Improved Hepatic Transduction, Reduced Systemic Vector Dissemination, and Long-Term Transgene Expression by Delivering Helper-Dependent Adenoviral Vectors into the Surgically Isolated Liver of Nonhuman Primates

  2006cited by this paper
• Helper-Dependent Adenoviral Vectors for Gene Therapy

  2005cited by this paper
• 904. High Efficiency Hepatic Transduction and Long-Term Transgene Expression by Delivering Helper-Dependent Adenoviral Vectors into the Surgically Isolated Liver of Nonhuman Primates

  2005cited by this paper
• Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection

  2005cited by this paper
• Persistent Gene Expression in Mouse Nasal Epithelia following Feline Immunodeficiency Virus-Based Vector Gene Transfer

  2005cited by this paper
• Lentivirus-Mediated Gene Transfer to the Central Nervous System: Therapeutic and Research Applications

  2005cited by this paper
• Failure of bone marrow to reconstitute lung epithelium.

  2005cited by this paper
• Targeted Gene Delivery by Intravenous Injection of Retroviral Vectors

  2005cited by this paper
• Viscoelastic gel formulations enhance airway epithelial gene transfer with viral vectors.

  2005cited by this paper
• Bone marrow-derived SP cells can contribute to the respiratory tract of mice in vivo

  2005cited by this paper
• Novel molecular approaches to cystic fibrosis gene therapy.

  2005cited by this paper
• VRX-496(VIRxSYS).

  2005cited by this paper
• 815. Aerosol Delivery of Helper-Dependent Adenoviral Vector into Nonhuman Primate Lungs Results in High Efficiency Pulmonary Transduction with Minimal Toxicity

  2005cited by this paper
• Gene Therapy Progress and Prospects: Development of improved lentiviral and retroviral vectors – design, biosafety, and production

  2005cited by this paper
• Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery.

  2005cited by this paper
• Bone marrow-derived mesenchymal stem cells in repair of the injured lung.

  2005cited by this paper
• Silencing and Variegation of Gammaretrovirus and Lentivirus Vectors

  2005cited by this paper
• Embryonic stem cells generate airway epithelial tissue.

  2005cited by this paper
• Correlation Between DNA Transfer and Cystic Fibrosis Airway Epithelial Cell Correction After Recombinant Adeno-Associated Virus Serotype 2 Gene Therapy

  2005cited by this paper
• Adeno-Associated Virus: A Ubiquitous Commensal of Mammals

  2005cited by this paper
• Characterization of wild-type and deltaF508 cystic fibrosis transmembrane regulator in human respiratory epithelia.

  2005cited by this paper
• Gene Transfer to Respiratory Epithelia with Lentivirus Pseudotyped with Jaagsiekte Sheep Retrovirus Envelope Glycoprotein

  2005cited by this paper
• Enhancing rAAV vector expression in the lung

  2005cited by this paper
• Cloning Researcher Says Work Is Flawed but Claims Results Stand

  2005cited by this paper
• Aerosol delivery of an enhanced helper‐dependent adenovirus formulation to rabbit lung using an intratracheal catheter

  2005cited by this paper
• Adult stem cells from bone marrow stroma differentiate into airway epithelial cells: potential therapy for cystic fibrosis.

  2005cited by this paper
• Intrapleural administration of a serotype 5 adeno-associated virus coding for α1-antitrypsin mediates persistent, high lung and serum levels of α1-antitrypsin

  2004cited by this paper
• Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial.

  2004cited by this paper
• Basal cells are a multipotent progenitor capable of renewing the bronchial epithelium.

  2004cited by this paper
• Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin.

  2004cited by this paper
• Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

  2004cited by this paper
• Advances in cystic fibrosis gene therapy

  2004cited by this paper
• Functional Characterization of a Recombinant Adeno- Associated Virus 5-Pseudotyped Cystic Fibrosis Transmembrane Conductance Regulator Vector

  2004cited by this paper
• Adeno-associated virus vectors integrate at chromosome breakage sites

  2004cited by this paper
• Distinct Classes of Proteasome-Modulating Agents Cooperatively Augment Recombinant Adeno-Associated Virus Type 2 and Type 5-Mediated Transduction from the Apical Surfaces of Human Airway Epithelia

  2004cited by this paper
• Fusion Proteins Consisting of Human Immunodeficiency Virus Type 1 Integrase and the Designed Polydactyl Zinc Finger Protein E2C Direct Integration of Viral DNA into Specific Sites

  2004cited by this paper
• DNA-dependent PK inhibits adeno-associated virus DNA integration

  2004cited by this paper
• Integrated Self-Inactivating Lentiviral Vectors Produce Full-Length Genomic Transcripts Competent for Encapsidation and Integration

  2004cited by this paper
• Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences

  2004cited by this paper
• Adenoviral Vectors for Gene Replacement Therapy

  2004cited by this paper
• Gene therapy progress and prospects: adenoviral vectors

  2003cited by this paper
• The impact of adenovirus infection on the immunocompromised host

  2003cited by this paper
• Human fetal trachea-SCID mouse xenografts: efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer.

  2003cited by this paper
• Targeting expression of a transgene to the airway surface epithelium using a ciliated cell-specific promoter.

  2003cited by this paper
• 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

  2003cited by this paper
• LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

  2003cited by this paper
• Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration

  2003cited by this paper
• Controlling integration specificity of a yeast retrotransposon

  2003cited by this paper
• Transplanted bone marrow regenerates liver by cell fusion

  2003cited by this paper
• Differentiation, cell fusion, and nuclear fusion during ex vivo repair of epithelium by human adult stem cells from bone marrow stroma

  2003cited by this paper
• Performance- and safety-enhanced lentiviral vectors containing the human interferon-beta scaffold attachment region and the chicken beta-globin insulator.

  2003cited by this paper
• Second-Strand Genome Conversion of Adeno-Associated Virus Type 2 (AAV-2) and AAV-5 Is Not Rate Limiting following Apical Infection of Polarized Human Airway Epithelia

  2003cited by this paper
• Reduced inflammation and improved airway expression using helper-dependent adenoviral vectors with a K18 promoter.

  2003cited by this paper
• Lentivirus Vectors Pseudotyped with Filoviral Envelope Glycoproteins Transduce Airway Epithelia from the Apical Surface Independently of Folate Receptor Alpha

  2003cited by this paper
• Performance- and safety-enhanced lentiviral vectors containing the human interferon-β scaffold attachment region and the chicken β-globin insulator

  2003cited by this paper
• Protection of Cftr knockout mice from acute lung infection by a helper-dependent adenoviral vector expressing Cftr in airway epithelia

  2003cited by this paper
• Cystic fibrosis

  2003cited by this paper
• A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.

  2003cited by this paper
• Mesenchymal stem cell engraftment in lung is enhanced in response to bleomycin exposure and ameliorates its fibrotic effects

  2003cited by this paper
• Improved system for helper-dependent adenoviral vector production.

  2003cited by this paper
• Safety and Efficiency of Modulating Paracellular Permeability to Enhance Airway Epithelial Gene Transfer In Vivo

  2003cited by this paper
• Safety of Local Delivery of Low- and Intermediate-Dose Adenovirus Gene Transfer Vectors to Individuals with a Spectrum of Morbid Conditions

  2002cited by this paper
• Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

  2002cited by this paper
• Integration site selection by lentiviruses: biology and possible control.

  2002cited by this paper
• Pancreatic stem cells

  2002cited by this paper
• Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

  2002cited by this paper
• HIV-1 integration in the human genome favors active genes and local hotspots.

  2002cited by this paper
• Recovery of Airway Cystic Fibrosis Transmembrane Conductance Regulator Function in Mice with Cystic Fibrosis After Single-Dose Lentivirus-Mediated Gene Transfer

  2002cited by this paper
• Gastrointestinal stem cells

  2002cited by this paper
• A Phase II, Double-Blind, Randomized, Placebo-Controlled Clinical Trial of tgAAVCF Using Maxillary Sinus Delivery in Patients with Cystic Fibrosis with Antrostomies

  2002cited by this paper
• Thixotropic solutions enhance viral-mediated gene transfer to airway epithelia.

  2002cited by this paper
• Identification of a novel population of muscle stem cells in mice

  2002cited by this paper
• Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

  2002cited by this paper
• Ubiquitination of both Adeno-Associated Virus Type 2 and 5 Capsid Proteins Affects the Transduction Efficiency of Recombinant Vectors

  2002cited by this paper
• Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors.

  2002cited by this paper
• Pluripotency of mesenchymal stem cells derived from adult marrow

  2002cited by this paper
• Regulation of gene expression in adeno-associated virus vectors in the brain.

  2002cited by this paper
• Derivation of Type II Alveolar Epithelial Cells from Murine Embryonic Stem Cells

  2002cited by this paper
• Effect Of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle

  2001cited by this paper
• Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo

  2001cited by this paper
• Adeno-Associated Virus Type 6 (AAV6) Vectors Mediate Efficient Transduction of Airway Epithelial Cells in Mouse Lungs Compared to That of AAV2 Vectors

  2001cited by this paper
• Targeting transgene expression for cystic fibrosis gene therapy.

  2001cited by this paper
• Systematic Determination of the Packaging Limit of Lentiviral Vectors

  2001cited by this paper
• Bone marrow cells regenerate infarcted myocardium

  2001cited by this paper
• Chromosomal Integration Pattern of a Helper-Dependent Minimal Adenovirus Vector with a Selectable Marker Inserted into a 27.4-Kilobase Genomic Stuffer

  2001cited by this paper
• Aerosol and Lobar Administration of a Recombinant Adenovirus to Individuals with Cystic Fibrosis. II. Transfection Efficiency in Airway Epithelium

  2001cited by this paper
• A Phase I Study of Aerosolized Administration of tgAAVCF to Cystic Fibrosis Subjects with Mild Lung Disease

  2001cited by this paper
• “Stealth” Adenoviruses Blunt Cell-Mediated and Humoral Immune Responses against the Virus and Allow for Significant Gene Expression upon Readministration in the Lung

  2001cited by this paper
• Somatic stem cell plasticity: current evidence and emerging concepts.

  2001cited by this paper
• Bone marrow-derived cells as progenitors of lung alveolar epithelium.

  2001cited by this paper
• Aerosol and Lobar Administration of a Recombinant Adenovirus to Individuals with Cystic Fibrosis. I. Methods, Safety, and Clinical Implications

  2001cited by this paper
• Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches.

  2001cited by this paper
• FIV Vector Systems

  2001cited by this paper
• Neovascularization of ischemic myocardium by human bone-marrow–derived angioblasts prevents cardiomyocyte apoptosis, reduces remodeling and improves cardiac function

  2001cited by this paper

• Diagnosis and treatment of cystic fibrosis in India: What is at stake for developing countries?

  2024cites this paper
• Advanced approaches to overcome biological barriers in respiratory and systemic routes of administration for enhanced nucleic acid delivery to the lung

  2023cites this paper
• Genome Editing for Cystic Fibrosis

  2023cites this paper
• Effective viral-mediated lung gene therapy: is airway surface preparation necessary?

  2022cites this paper
• Breaching the Delivery Barrier: Chemical and Physical Airway Epithelium Disruption Strategies for Enhancing Lentiviral-Mediated Gene Therapy

  2021cites this paper
• Gene therapy for cystic fibrosis: new tools for precision medicine

  2021cites this paper
• Overcoming Immunological Challenges to Helper-Dependent Adenoviral Vector-Mediated Long-Term CFTR Expression in Mouse Airways

  2020cites this paper
• Detargeting Lentiviral-Mediated CFTR Expression in Airway Basal Cells Using miR-106b

  2020cites this paper
• Aerosol Inhalation-mediated Delivery of an Adeno-associated Virus 5-expressed Antagonistic Interleukin-4 Mutant Ameliorates Experimental Murine Asthma.

  2019cites this paper
• Efficient Nonviral Transfection of Human Bone Marrow Mesenchymal Stromal Cells Shown Using Placental Growth Factor Overexpression

  2018cites this paper
• Long-Term Expression of the Human CFTR Gene in Mouse Airway via Helper-Dependent Adenoviral Vector Delivery and Transient Immunosuppression

  2016cites this paper
• Testing gene therapy vectors in human primary nasal epithelial cultures

  2015cites this paper
• Investigation and application of novel adeno-associated viral vectors for Cystic Fibrosis gene therapy

  2015cites this paper
• Efficient intratracheal delivery of airway epithelial cells in mice and pigs.

  2015cites this paper
• Lung gene therapy—How to capture illumination from the light already present in the tunnel

  2014cites this paper
• In Utero Lung Gene Transfer Using Adeno-Associated Viral and Lentiviral Vectors in Mice

  2014cites this paper
• Expert opinion in biological therapy: update on developments in lung gene transfer

  2013cites this paper
• Viral‐Mediated Delivery of shRNA and miRNA

  2013cites this paper
• Gene Therapy Perspectives Against Diseases of the Respiratory System

  2013cites this paper
• Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy

  2013cites this paper
• Vectors following Intranasal Delivery Response to Helper-Dependent Adenoviral Characterization of Pulmonary T Cell

  2013cites this paper
• Interaction between Macrophages and Epithelial Cells in Innate Immune Responses against Adenoviral Vectors

  2012cites this paper
• Differentiated swine airway epithelial cell cultures for the investigation of influenza A virus infection and replication

  2012cites this paper
• Hematopoietic Stem/Progenitor Cells Express Functional Mitochondrial Energy-Dependent Cystic Fibrosis Transmembrane Conductance Regulator

  2012cites this paper
• Cell-Based Therapies for Lung Vascular Diseases

  2012cites this paper
• Treatment Options in Motor Neuron Disease: Amyotrophic Lateral Sclerosis and Spinal Muscular Atrophy

  2012cites this paper
• Strategies for improving adeno-associated viral infection of airway epithelial cells

  2012cites this paper
• Progress in gene and cell therapy for cystic fibrosis lung disease.

  2012cites this paper
• Vector Specific Tolerance Induction for Airwary Gene Therapy

  2012cites this paper
• Gene Regulatable Lentiviral Vector System

  2011cites this paper
• Readministration of helper-dependent adenoviral vectors to mouse airway mediated via transient immunosuppression

  2011cites this paper
• Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector

  2011cites this paper
• Augmentation of rat skin flap viability by relaxin‐expressing adenovirus

  2011cites this paper
• Subretinal gene delivery using helper-dependent adenoviral vectors

  2011cites this paper
• Gene and cell therapy for cystic fibrosis: from bench to bedside.

  2011cites this paper
• Gene therapy: light is finally in the tunnel

  2011influential citation
• Keratinocyte growth factor (KGF) gene therapy mediated by an attenuated form of Salmonella typhimurium ameliorates radiation induced pulmonary injury in rats.

  2011cites this paper
• Adeno-associated virus serotype 9-mediated pulmonary transgene expression: effect of mouse strain, animal gender and lung inflammation

  2011cites this paper
• Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR−/− mice

  2011cites this paper
• Stem cells and cell therapies in lung biology and lung diseases.

  2011cites this paper
• Tolerogenic dendritic cells and their potential applications

  2011cites this paper
• Sperimentazioni cliniche di terapia genica

  2011cites this paper
• Therapie höhergradiger Gliome mit dem onkolytischen Stamm MTH-68/H des Newcastle-Disease-Virus in der pädiatrischen Onkologie

  2010cites this paper
• Polarity Influences the Efficiency of Recombinant Adenoassociated Virus Infection in Differentiated Airway Epithelia

  2010cites this paper
• Adenoviral vectors stimulate innate immune responses in macrophages through cross-talk with epithelial cells.

  2010cites this paper
• Lentiviral Vectors and Cystic Fibrosis Gene Therapy

  2010cites this paper
• Viral vectors for cystic fibrosis gene therapy: What does the future hold?

  2010cites this paper
• Development of an inflammation‐inducible gene expression system using helper‐dependent adenoviral vectors

  2010cites this paper
• Clinical Applications of Gene Therapy

  2010cites this paper
• Identification of ebola glycoprotein mutants that exhibit increased transduction efficiency

  2009cites this paper
• Functional Cystic Fibrosis Transmembrane Conductance Regulator Expression in Cystic Fibrosis Airway Epithelial Cells by AAV6.2-Mediated Segmental Trans-Splicing

  2009cites this paper
• New Genetic and Pharmacological Treatments for Cystic Fibrosis

  2009cites this paper
• Engineered adenovirus serotypes for overcoming anti-vector immunity

  2009cites this paper
• Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro.

  2009cites this paper
• Directed evolution of adeno-associated virus to an infectious respiratory virus

  2009cites this paper
• Involvement of glycosaminoglycans in vesicular stomatitis virus G glycoprotein pseudotyped lentiviral vector‐mediated gene transfer into airway epithelial cells

  2008cites this paper
• Cystic fibrosis: recent advances in lung-directed therapies

  2008cites this paper
• Hydrator therapies for cystic fibrosis lung disease

  2008cites this paper
• Characterization of Pulmonary T Cell Response to Helper-Dependent Adenoviral Vectors following Intranasal Delivery1

  2008cites this paper
• Gene therapy for cystic fibrosis

  2008cites this paper
• Noninvasive Molecular Neuroimaging Using Reporter Genes: Part II, Experimental, Current, and Future Applications

  2008cites this paper
• Hepatocyte growth factor enhances engraftment and function of nonhuman primate islets.

  2008cites this paper
• Lentiviral Vector for Gene Transfer A Versatile Tool for Regulated Gene Expression, Gene Silencing and Progenitor Cell Therapies

  2008cites this paper
• Stem cells and cell therapies in lung biology and lung diseases.

  2008cites this paper
• Gene Therapy for Cystic Fibrosis

  2008cites this paper
• Hepatocyte Growth Factor Enhances Engraftment and Function of Nonhuman Primate Islets

  2008cites this paper
• Gene Transfer in Differentiated Primary Rat Tracheal Epithelial Cells by Non-Viral Vectors

  2007cites this paper
• Gene and cell therapy for cystic fibrosis.

  2006cites this paper
• Gene Delivery to the Airway

  1999cites this paper