(Q36712185)

15 August 2017

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title

Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis (English)

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15 August 2017

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Paul B McCray

4

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author name string

Terence R Flotte

1

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15 August 2017

Philip Ng

2

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15 August 2017

Doug E Dylla

3

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15 August 2017

Guoshun Wang

5

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15 August 2017

Jay K Kolls

6

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15 August 2017

Jim Hu

7

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15 August 2017

publication date

1 February 2007

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15 August 2017

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15 August 2017

volume

15

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15 August 2017

issue

2

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15 August 2017

page(s)

229-241

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Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA

15 August 2017

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7 January 2021

Safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lungs of nonhuman primates.

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7 January 2021

In vivo evaluation of the safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lung.

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7 January 2021

Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo

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7 January 2021

Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression

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7 January 2021

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7 January 2021

Toxicological comparison of E2a-deleted and first-generation adenoviral vectors expressing alpha1-antitrypsin after systemic delivery

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7 January 2021

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7 January 2021

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7 January 2021

Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a.

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7 January 2021

Transfer of the CFTR Gene to the Lung of Nonhuman Primates with E1-Deleted, E2a-Defective Recombinant Adenoviruses: A Preclinical Toxicology Study

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7 January 2021

Helper-dependent adenoviral vectors for gene therapy

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7 January 2021

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7 January 2021

Reduced inflammation and improved airway expression using helper-dependent adenoviral vectors with a K18 promoter.

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7 January 2021

Targeting transgene expression to airway epithelia and submucosal glands, prominent sites of human CFTR expression

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7 January 2021

Targeting transgene expression for cystic fibrosis gene therapy

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

"Stealth" adenoviruses blunt cell-mediated and humoral immune responses against the virus and allow for significant gene expression upon readministration in the lung

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7 January 2021

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7 January 2021

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7 January 2021

"Sero-switch" adenovirus-mediated in vivo gene transfer: circumvention of anti-adenovirus humoral immune defenses against repeat adenovirus vector administration by changing the adenovirus serotype

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7 January 2021

Adeno-associated virus: a ubiquitous commensal of mammals

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7 January 2021

Detection of adeno-associated virus (AAV)-specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells

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7 January 2021

The cryptic life style of adeno-associated virus

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7 January 2021

Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells

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7 January 2021

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

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7 January 2021

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

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7 January 2021

Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors

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7 January 2021

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

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7 January 2021

Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

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7 January 2021

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7 January 2021

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7 January 2021

Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector

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7 January 2021

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease

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7 January 2021

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

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7 January 2021

A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis

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7 January 2021

Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease

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7 January 2021

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7 January 2021

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

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7 January 2021

Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy

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7 January 2021

Polarity influences the efficiency of recombinant adenoassociated virus infection in differentiated airway epithelia.

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7 January 2021

Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus.

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7 January 2021

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7 January 2021

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7 January 2021

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

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7 January 2021

Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors.

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7 January 2021

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

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7 January 2021

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

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7 January 2021

Enhancing rAAV vector expression in the lung

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7 January 2021

Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin

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7 January 2021

Cell proliferation in bronchial epithelium and submucosal glands of cystic fibrosis patients

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7 January 2021

Organization of adeno-associated virus DNA in latently infected Detroit 6 cells

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7 January 2021

Site-specific integration by adeno-associated virus

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7 January 2021

Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter

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7 January 2021

Targeted integration of adeno-associated virus (AAV) into human chromosome 19

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7 January 2021

Adeno-associated virus: integration at a specific chromosomal locus

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7 January 2021

Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle

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7 January 2021

DNA-dependent PK inhibits adeno-associated virus DNA integration

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7 January 2021

Adeno-associated virus vectors integrate at chromosome breakage sites

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7 January 2021

A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells

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7 January 2021

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

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7 January 2021

Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect

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7 January 2021

Systematic determination of the packaging limit of lentiviral vectors

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7 January 2021

Lentiviral vectors for gene therapy of cystic fibrosis.

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7 January 2021

Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer

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7 January 2021

Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors

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7 January 2021

Gene transfer vectors derived from equine infectious anemia virus

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7 January 2021

Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production.

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7 January 2021

Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

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7 January 2021

Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

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7 January 2021

Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration

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7 January 2021

Development of an epithelium-specific expression cassette with human DNA regulatory elements for transgene expression in lung airways

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7 January 2021

Targeting expression of a transgene to the airway surface epithelium using a ciliated cell-specific promoter

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7 January 2021

Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo.

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7 January 2021

Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Correction of the apical membrane chloride permeability defect in polarized cystic fibrosis airway epithelia following retroviral-mediated gene transfer

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7 January 2021

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

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7 January 2021

Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches

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7 January 2021

Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery

1 reference

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7 January 2021

Human fetal trachea-SCID mouse xenografts: efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer.

1 reference

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7 January 2021

Enhanced epithelial gene transfer by modulation of tight junctions with sodium caprate.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo

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7 January 2021

Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce airway epithelia from the apical surface independently of folate receptor alpha

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7 January 2021

Retrovirus vectors bearing jaagsiekte sheep retrovirus Env transduce human cells by using a new receptor localized to chromosome 3p21.3.

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7 January 2021

Gene transfer to respiratory epithelia with lentivirus pseudotyped with Jaagsiekte sheep retrovirus envelope glycoprotein

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7 January 2021

Persistent gene expression in mouse nasal epithelia following feline immunodeficiency virus-based vector gene transfer

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7 January 2021

Influenza M2 envelope protein augments avian influenza hemagglutinin pseudotyping of lentiviral vectors

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7 January 2021

Viscoelastic Gel Formulations Enhance Airway Epithelial Gene Transfer with Viral Vectors

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7 January 2021

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

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7 January 2021

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

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7 January 2021

Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences

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7 January 2021

HIV-1 integration in the human genome favors active genes and local hotspots

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Transcription start regions in the human genome are favored targets for MLV integration

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Silencing and variegation of gammaretrovirus and lentivirus vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Controlling integration specificity of a yeast retrotransposon

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Performance- and safety-enhanced lentiviral vectors containing the human interferon-beta scaffold attachment region and the chicken beta-globin insulator

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Targeted gene delivery by intravenous injection of retroviral vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Thixotropic solutions enhance viral-mediated gene transfer to airway epithelia.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Derivation of type II alveolar epithelial cells from murine embryonic stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Embryonic stem cells generate airway epithelial tissue

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Neuroepithelial bodies of pulmonary airways serve as a reservoir of progenitor cells capable of epithelial regeneration

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

A clonogenic common myeloid progenitor that gives rise to all myeloid lineages

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Cytological Identification of Radiation-Chimæras

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Identification of clonogenic common lymphoid progenitors in mouse bone marrow

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Somatic stem cell plasticity: current evidence and emerging concepts.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Dystrophin expression in the mdx mouse restored by stem cell transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Regeneration of ischemic cardiac muscle and vascular endothelium by adult stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Pluripotency of mesenchymal stem cells derived from adult marrow

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Neovascularization of ischemic myocardium by human bone-marrow-derived angioblasts prevents cardiomyocyte apoptosis, reduces remodeling and improves cardiac function

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Multi-organ, multi-lineage engraftment by a single bone marrow-derived stem cell

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Bone marrow cells regenerate infarcted myocardium

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Bone marrow as a potential source of hepatic oval cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Assessment of cystic fibrosis transmembrane conductance regulator (CFTR) activity in CFTR-null mice after bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Limited restoration of cystic fibrosis lung epithelium in vivo with adult bone marrow-derived cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Failure of bone marrow to reconstitute lung epithelium

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Bone marrow-derived SP cells can contribute to the respiratory tract of mice in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Multilineage potential of adult human mesenchymal stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Marrow stromal cells as stem cells for nonhematopoietic tissues

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Hepatocytes from non-hepatic adult stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Pancreatic stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Gastrointestinal stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Mammalian neural stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Circulating skeletal stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Mesenchymal stem cell engraftment in lung is enhanced in response to bleomycin exposure and ameliorates its fibrotic effects

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Marrow stromal cells as a source of progenitor cells for nonhematopoietic tissues in transgenic mice with a phenotype of osteogenesis imperfecta

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Bone marrow-derived mesenchymal stem cells in repair of the injured lung

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Basal cells are a multipotent progenitor capable of renewing the bronchial epithelium

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Viable offspring derived from fetal and adult mammalian cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Embryonic and somatic cell cloning.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Transplanted bone marrow regenerates liver by cell fusion

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Stem cells. Cloning researcher says work is flawed but claims results stand

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300002

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300002

1 reference

15 August 2017

1 reference