(Q28708928)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

title

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012 (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

0 references

0 references

0 references

0 references

2

1 reference

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26 February 2020

Katherine A. High

13

1 reference

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26 February 2020

Samuel G. Jacobson

14

1 reference

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26 February 2020

James Wilson

21

1 reference

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26 February 2020

author name string

Marina O'Reilly

1

1 reference

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26 February 2020

Jeffrey Bartlett

3

1 reference

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26 February 2020

Janet Benson

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

Philip J Brooks

5

1 reference

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26 February 2020

Barry J Byrne

6

1 reference

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26 February 2020

Carlos Camozzi

7

1 reference

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26 February 2020

Kenneth Cornetta

8

1 reference

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26 February 2020

Ronald G Crystal

9

1 reference

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26 February 2020

Yuman Fong

10

1 reference

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26 February 2020

Linda Gargiulo

11

1 reference

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26 February 2020

Rashmi Gopal-Srivastava

12

1 reference

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26 February 2020

Robert C Jambou

15

1 reference

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26 February 2020

Maureen Montgomery

16

1 reference

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26 February 2020

Eugene Rosenthal

17

1 reference

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26 February 2020

R Jude Samulski

18

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

Sonia I Skarlatos

19

1 reference

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26 February 2020

Brian Sorrentino

20

1 reference

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26 February 2020

Yun Xie

22

1 reference

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26 February 2020

Jacqueline Corrigan-Curay

23

1 reference

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26 February 2020

language of work or name

English

0 references

publication date

1 April 2013

1 reference

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26 February 2020

1 reference

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26 February 2020

volume

24

1 reference

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26 February 2020

issue

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

page(s)

355-362

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

26 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=3631014

Efficacy of gene therapy for X-linked severe combined immunodeficiency

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

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

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Gene therapy for primary immunodeficiencies: Part 2.

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

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

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

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

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Gene therapy for primary immunodeficiency

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients

27 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

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

27 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3631014

27 November 2018

Identifiers

DOI

10.1089/HUM.2013.064

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

1 reference

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23517518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

PubMed publication ID

23517518

2 references