(Q35180848)

7 August 2017

title

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

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7 August 2017

Chengwen Li

1

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7 August 2017

Pingjie Xiao

2

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7 August 2017

Steven James Gray

3

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7 August 2017

Marc Scott Weinberg

4

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7 August 2017

R Jude Samulski

5

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7 August 2017

language of work or name

English

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publication date

15 August 2011

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Proceedings of the National Academy of Sciences of the United States of America

7 August 2017

published in

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7 August 2017

volume

108

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7 August 2017

issue

34

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7 August 2017

page(s)

14258-14263

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Regulation of innate immune responses by autophagy-related proteins

7 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

AAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T cells

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

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

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

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

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

The role of autophagy in alpha-1-antitrypsin deficiency: a specific cellular response in genetic diseases associated with aggregation-prone proteins

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

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

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Gene therapy for human alpha1-antitrypsin deficiency in an animal model using SV40-derived vectors

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Unfolding the role of protein misfolding in neurodegenerative diseases

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3161599

Adeno-associated virus vectors: potential applications for cancer gene therapy

28 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1109522108

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/10.1073%2FPNAS.1109522108

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1109522108

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1109522108

Clades of Adeno-associated viruses are widely disseminated in human tissues.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1109522108

21 January 2018

Identifiers

DOI

10.1073/PNAS.1109522108

1 reference

7 August 2017

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7 August 2017

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