Long-term safety and efficacy of factor IX gene therapy in hemophilia B. (Q34783922)

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23 February 2020

title

Long-term safety and efficacy of factor IX gene therapy in hemophilia B (English)

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23 February 2020

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coagulation factor IX

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5

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Marco Della Peruta

7

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Katherine A. High

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3

Edward G D Tuddenham

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1

Amit C Nathwani

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Federico Mingozzi

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Andrew M Davidoff

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23 February 2020

author name string

Ulreke M Reiss

2

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Cecilia Rosales

4

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Jenny McIntosh

6

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Elsa Lheriteau

8

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Nishal Patel

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Deepak Raj

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Anne Riddell

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Jun Pie

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Savita Rangarajan

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David Bevan

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Michael Recht

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Yu-Min Shen

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Kathleen G Halka

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Etiena Basner-Tschakarjan

18

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James Allay

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Mark A Kay

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Catherine Y C Ng

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Junfang Zhou

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Maria Cancio

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Christopher L Morton

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John T Gray

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Deokumar Srivastava

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Arthur W Nienhuis

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language of work or name

English

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publication date

1 November 2014

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The New England Journal of Medicine

23 February 2020

published in

1 reference

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23 February 2020

volume

371

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23 February 2020

issue

21

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23 February 2020

page(s)

1994-2004

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Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods

23 February 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

The hope and reality of long-acting hemophilia products

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

Epidemiology of coagulation disorders

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

Randomized, multicenter trial comparing tacrolimus plus mycophenolate mofetil to tacrolimus plus steroids in hepatitis C virus-positive recipients of living donor liver transplantation.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

Walk a mile in the moccasins of people with haemophilia

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

CD8( ) T-cell responses to adeno-associated virus capsid in humans

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4278802

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=4278802

26 October 2018

Identifiers

DOI

10.1056/NEJMOA1407309

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25409372 AND SRC:MED&resulttype=core&format=json

23 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25409372 AND SRC:MED&resulttype=core&format=json

23 February 2020

PubMed publication ID

25409372

1 reference