Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. (Q36438711)

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English	Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.	scientific article

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7 November 2019

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1 (English)

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7 November 2019

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coagulation factor IX

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Joerg Schuettrumpf

2

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7 November 2019

Federico Mingozzi

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Federico Mingozzi

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7 November 2019

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Valder R Arruda

1

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7 November 2019

Roland W Herzog

3

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7 November 2019

Timothy C Nichols

4

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7 November 2019

Nancy Robinson

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7 November 2019

Yasmin Lotfi

6

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7 November 2019

Weidong Xiao

8

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7 November 2019

Linda B Couto

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7 November 2019

Katherine A High

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7 November 2019

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11 September 2003

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7 November 2019

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7 November 2019

103

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7 November 2019

1

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7 November 2019

85-92

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7 November 2019

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

Enhancement of muscle gene delivery with pseudotyped adeno-associated virus type 5 correlates with myoblast differentiation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

Localization dose and time of antigens determine immune reactivity

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

Gene therapy vectors based on adeno-associated virus type 1.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

Adeno-associated virus vectors can be efficiently produced without helper virus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

Infectious Clones and Vectors Derived from Adeno-Associated Virus (AAV) Serotypes Other Than AAV Type 2

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

Canine hemophilia B resulting from a point mutation with unusual consequences

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

30 September 2017

A coagulation factor IX-deficient mouse model for human hemophilia B

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

28 June 2018

Molecular cloning of a cDNA encoding canine factor IX.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

28 June 2018

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4704086

21 September 2018

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

1 reference

https://pubmed.ncbi.nlm.nih.gov/12969984

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/12969984

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony

1 reference

https://pubmed.ncbi.nlm.nih.gov/12969984

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1182/BLOOD-2003-05-1446

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Europe PubMed Central

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7 November 2019

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Europe PubMed Central

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7 November 2019

PubMed publication ID

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Europe PubMed Central

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7 November 2019

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