Current status of gene therapy for α-1 antitrypsin deficiency (Q38264336)

1 September 2017

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title

Current status of gene therapy for α-1 antitrypsin deficiency (English)

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1 September 2017

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Heather S Loring

1

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PubMed ID

25363251

1 September 2017

Terence R Flotte

series ordinal

2

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PubMed ID

25363251

1 September 2017

language of work or name

English

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publication date

3 November 2014

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Expert Opinion on Biological Therapy

1 September 2017

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1 September 2017

15

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1 September 2017

329-336

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1 September 2017

3

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The electrophoretic α1-globulin pattern of serum in α1-antitrypsin deficiency. 1963.

1 September 2017

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

The Electrophoretic α;1-Globulin Pattern of Serum in α;1-Antitrypsin Deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

The alpha 1-antitrypsin gene and its deficiency states

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

The alpha 1-antitrypsin gene and its mutations. Clinical consequences and strategies for therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

A review of α1-antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Proteolytic Enzymes, their Inhibitors and Lung Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Alpha1-antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

The mechanism of Z alpha 1-antitrypsin accumulation in the liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Alpha 1-antitrypsin deficiency deaths in the United States from 1979-1991. An analysis using multiple-cause mortality data

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Asthma and allergy in alpha-1 antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Airway disease in alpha-1 antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Loop-sheet polymerization: the structural basis of Z alpha 1-antitrypsin accumulation in the liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Alpha-1-antitrypsin: an ignored protein in understanding liver disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Danazol-induced augmentation of serum alpha 1-antitrypsin levels in individuals with marked deficiency of this antiprotease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

The circulating proteinase inhibitor α-1 antitrypsin regulates neutrophil degranulation and autoimmunity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Fate of aerosolized recombinant DNA-produced alpha 1-antitrypsin: use of the epithelial surface of the lower respiratory tract to administer proteins of therapeutic importance

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Strategies for aerosol therapy of alpha 1-antitrypsin deficiency by the aerosol route

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Gene-based therapy for alpha-1 antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: comparison with protein therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Adeno-associated virus: a ubiquitous commensal of mammals

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Clinical gene therapy using recombinant adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

DNA-dependent PK inhibits adeno-associated virus DNA integration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.978854

10.1517/14712598.2015.978854

21 January 2018

Identifiers

DOI

1 reference

1 September 2017

1 reference

PubMed ID

25363251