Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial (Q34843808)

4 August 2017

title

Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial (English)

1 reference

https://api.crossref.org/works/10.1089/HUM.2010.202

4 August 2017

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial (English)

1 reference

27 November 2024

0 references

0 references

Good manufacturing practice

1 reference

Andrew M Davidoff

1 reference

4 August 2017

Susan Sleep

1 reference

4 August 2017

Scott Long

1 reference

4 August 2017

Rob Clark

1 reference

4 August 2017

Gael Carney

1 reference

4 August 2017

Paolo Fagone

1 reference

4 August 2017

James A. Allay

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

27 November 2024

David M. Tillman

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

27 November 2024

Jenny H. McIntosh

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

27 November 2024

Arthur W. Nienhuis

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

27 November 2024

Amit C. Nathwani

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

27 November 2024

John T. Gray

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

27 November 2024

language of work or name

English

1 reference

National Center for Biotechnology Information

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/esummary.fcgi?db=pubmed&retmode=json&id=21410419

27 November 2024

publication date

1 May 2011

1 reference

4 August 2017

May 2011

1 reference

National Center for Biotechnology Information

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/esummary.fcgi?db=pubmed&retmode=json&id=21410419

full work available at URL

27 November 2024

https://europepmc.org/articles/pmc3081439?pdf=render

file format

Portable Document Format

online access status

open access

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

https://europepmc.org/articles/PMC3081439

27 November 2024

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

https://europepmc.org/articles/PMC3081439?pdf=render

27 November 2024

file format

Portable Document Format

online access status

open access

content deliverer

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

https://doi.org/10.1089/hum.2010.202

27 November 2024

online access status

open access

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A21410419

http://www.liebertpub.com/doi/full-xml/10.1089/hum.2010.202

27 November 2024

1 reference

https://api.crossref.org/works/10.1089/HUM.2010.202

http://www.liebertpub.com/doi/pdf/10.1089/hum.2010.202

27 November 2024

1 reference

https://api.crossref.org/works/10.1089/HUM.2010.202

27 November 2024

1 reference

4 August 2017

1 reference

4 August 2017

1 reference

4 August 2017

page(s)

595-604

1 reference

A Versatile Adeno-Associated Virus Vector Producer Cell Line Method for Scalable Vector Production of Different Serotypes

4 August 2017

cites work

1 reference

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8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

8 July 2018

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8 July 2018

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

8 July 2018

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Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

8 July 2018

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Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery.

8 July 2018

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Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

8 July 2018

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Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

8 July 2018

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Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

8 July 2018

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Quantification of adeno-associated virus particles and empty capsids by optical density measurement

8 July 2018

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

8 July 2018

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A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5.

8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson’s disease in rats

8 July 2018

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8 July 2018

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8 July 2018

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8 July 2018

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High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3' gamma globin gene regulatory element and integrates as tandem copies in erythroid cells

8 July 2018

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8 July 2018

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8 July 2018

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High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

8 July 2018

1 reference

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Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

26 October 2018

1 reference

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Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

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

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

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

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Maxillary sinusitis as a surrogate model for CF gene therapy clinical trials in patients with antrostomies

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

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

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

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

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Recombinant adeno-associated virus for muscle directed gene therapy

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3081439

Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

26 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21410419

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

https://api.crossref.org/works/10.1089/HUM.2010.202

African-American ethnicity is associated with higher plasma levels of D-dimer in adults with hypertension

27 November 2024

1 reference

https://api.crossref.org/works/10.1089/HUM.2010.202

Increased apoptosis of Huntington disease lymphoblasts associated with repeat length-dependent mitochondrial depolarization

27 November 2024

1 reference

https://api.crossref.org/works/10.1089/HUM.2010.202

27 November 2024

Identifiers

DOI

10.1089/HUM.2010.202

1 reference

4 August 2017

1 reference

4 August 2017

1 reference