(Q39675326)

13 September 2017

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy (English)

1 reference

13 September 2017

0 references

1

Nisha Nair

1 reference

13 September 2017

Melvin Y Rincon

2

1 reference

13 September 2017

Hanneke Evens

3

1 reference

13 September 2017

Shilpita Sarcar

4

1 reference

13 September 2017

Sumitava Dastidar

5

1 reference

13 September 2017

Emira Samara-Kuko

6

1 reference

13 September 2017

Omid Ghandeharian

7

1 reference

13 September 2017

Hiu Man Viecelli

8

1 reference

13 September 2017

Beat Thöny

9

1 reference

13 September 2017

Pieter De Bleser

10

1 reference

13 September 2017

Thierry VandenDriessche

11

1 reference

13 September 2017

Marinee K Chuah

12

1 reference

13 September 2017

language of work or name

English

0 references

publication date

17 March 2014

1 reference

13 September 2017

1 reference

13 September 2017

123

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13 September 2017

20

1 reference

13 September 2017

3195-3199

1 reference

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

13 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

X-linked thrombophilia with a mutant factor IX (factor IX Padua).

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

D-dimer antigen: current concepts and future prospects

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

A factor IX-deficient mouse model for hemophilia B gene therapy

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

A distance difference matrix approach to identifying transcription factors that regulate differential gene expression

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice

15 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

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

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

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

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4023424

10.1182/BLOOD-2013-10-534032

21 August 2018

Identifiers

DOI

1 reference

13 September 2017

1 reference

13 September 2017

PubMed publication ID

24637359

1 reference