Gene-based therapy for alpha-1 antitrypsin deficiency (Q38092873)

28 August 2017

Gene-based therapy for alpha-1 antitrypsin deficiency (English)

1 reference

28 August 2017

Christian Mueller

1

1 reference

PubMed ID

23527792

28 August 2017

Terence R Flotte

series ordinal

2

1 reference

PubMed ID

23527792

28 August 2017

language of work or name

English

0 references

publication date

1 March 2013

1 reference

COPD: Journal of Chronic Obstructive Pulmonary Disease

28 August 2017

1 reference

28 August 2017

10 Suppl 1

1 reference

28 August 2017

44-49

1 reference

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

28 August 2017

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

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

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Transplantation of genetically modified autologous hepatocytes into nonhuman primates: feasibility and short-term toxicity.

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

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

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

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/10.3109%2F15412555.2013.764978

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

DNA-dependent PK inhibits adeno-associated virus DNA integration

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

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/10.3109%2F15412555.2013.764978

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

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/10.3109%2F15412555.2013.764978

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/10.3109%2F15412555.2013.764978

AAVrh.10-mediated genetic delivery of bevacizumab to the pleura to provide local anti-VEGF to suppress growth of metastatic lung tumors

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Ribozyme-mediated specific gene replacement of the alpha1-antitrypsin gene in human hepatoma cells

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.3109%2F15412555.2013.764978

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/10.3109%2F15412555.2013.764978

10.3109/15412555.2013.764978

21 January 2018

Identifiers

DOI

1 reference

28 August 2017

1 reference

PubMed ID

23527792