Update on clinical gene therapy for hemophilia (Q90595648)

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scientific article published on 17 December 2018

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English	Update on clinical gene therapy for hemophilia	scientific article published on 17 December 2018

Statements

instance of

scholarly article

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

title

Update on clinical gene therapy for hemophilia (English)

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

main subject

hemophilia

1 reference

based on heuristic

inferred from title

author

Roland W Herzog

series ordinal

2

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

David M Markusic

series ordinal

3

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

author name string

George Q Perrin

series ordinal

1

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

publication date

17 December 2018

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stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

published in

Blood

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

volume

133

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

issue

5

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

page(s)

407-414

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

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The molecular basis of haemophilia A and B.

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12 December 2020

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ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES

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12 December 2020

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

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12 December 2020

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Cellular immune responses in hemophilia: why do inhibitors develop in some, but not all hemophiliacs?

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12 December 2020

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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

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12 December 2020

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

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12 December 2020

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Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

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12 December 2020

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Inhibitor development in haemophilia B: an orphan disease in need of attention

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12 December 2020

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AAV vector integration sites in mouse hepatocellular carcinoma.

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12 December 2020

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AAV vectors and tumorigenicity

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12 December 2020

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Antioxidants reduce endoplasmic reticulum stress and improve protein secretion

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12 December 2020

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Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells

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12 December 2020

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

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12 December 2020

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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

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12 December 2020

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Assessing the potential for AAV vector genotoxicity in a murine model

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12 December 2020

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Induction of tolerance to factor VIII by transient co-administration with rapamycin.

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PubMed

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12 December 2020

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Immune tolerance induction in haemophilia: evidence and the way forward

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

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Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver

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12 December 2020

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Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy

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12 December 2020

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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

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12 December 2020

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

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Overcoming preexisting humoral immunity to AAV using capsid decoys

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Toward optimal therapy for inhibitors in hemophilia

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell-dependent induction of CD4+CD25+FoxP3+ Treg.

1 reference

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

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Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Delivery and therapeutic applications of gene editing technologies ZFNs, TALENs, and CRISPR/Cas9.

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene therapy for immune tolerance induction in hemophilia with inhibitors

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV.

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Reply to "Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV"

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Genome-editing technologies for gene correction of hemophilia

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

2017 Clinical trials update: Innovations in hemophilia therapy

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Combination therapy for inhibitor reversal in haemophilia A using monoclonal anti-CD20 and rapamycin.

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy.

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The Balance between CD8+ T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models.

1 reference

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Therapeutic gene editing: delivery and regulatory perspectives

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene Therapy for Hemophilia

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8+ T Cells

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Complexity of immune responses to AAV transgene products - Example of factor IX.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Advances in Gene Therapy for Haemophilia

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Unraveling the complex story of immune responses to AAV vectors trial after trial

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene Therapy for Hemophilia.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in children affected by Pompe Disease

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A Robust System for Production of Superabundant VP1 Recombinant AAV Vectors.

1 reference

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Platelet-Targeted Gene Therapy for Hemophilia.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A.

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Liver induced transgene tolerance with AAV vectors

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Novel therapeutics for hemophilia and other bleeding disorders

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Haemophilia gene therapy: From trailblazer to gamechanger

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Advances and innovations in haemophilia treatment

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

1 reference

stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/30559260

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

DOI

10.1182/BLOOD-2018-07-820720

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

PMC publication ID

6356985

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

PubMed publication ID

30559260

1 reference

stated in

Europe PubMed Central

PMC publication ID

6356985

reference URL

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30559260%20AND%20SRC:MED&resulttype=core&format=json

retrieved

14 April 2020

 

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