Safety of adeno-associated virus gene therapy vectors: a current evaluation (Q35194547)

7 August 2017

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title

Safety of adeno-associated virus gene therapy vectors: a current evaluation (English)

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7 August 2017

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Paul E Monahan

1

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7 August 2017

Karin Jooss

series ordinal

2

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7 August 2017

Mark S Sands

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3

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7 August 2017

language of work or name

English

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publication date

1 May 2002

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Expert Opinion on Drug Safety

7 August 2017

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7 August 2017

1

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7 August 2017

1

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7 August 2017

79-91

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Gene therapy. Viral vectors still pack surprises

7 August 2017

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https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus (AAV) site-specific recombination does not require a Rep-dependent origin of replication within the AAV terminal repeat

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus site-specifically integrates into a muscle-specific DNA region

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Building a better vector: the manipulation of AAV virions

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Real-time single-molecule imaging of the infection pathway of an adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Intracellular trafficking of adeno-associated virus vectors: routing to the late endosomal compartment and proteasome degradation

21 January 2018

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https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Viral receptors and vector purification: new approaches for generating clinical-grade reagents

21 January 2018

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https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Production and purification of recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

High-titer, wild-type free recombinant adeno-associated virus vector production using intron-containing helper plasmids

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adenovirus hexon protein is a potent adjuvant for activation of a cellular immune response

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration

21 January 2018

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https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Chromosomal effects of adeno-associated virus vector integration

21 January 2018

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https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Site-specific integration of an adeno-associated virus vector plasmid mediated by regulated expression of rep based on Cre-loxP recombination

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus type 2 nonstructural protein Rep78 suppresses translation in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Kinetics of recombinant adeno-associated virus-mediated gene transfer

21 January 2018

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https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Persistent expression of canine factor IX in hemophilia B canines

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Evidence for infection of the human embryo with adeno-associated virus in pregnancy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Evaluating the potential of germ line transmission after intravenous administration of recombinant adenovirus in the C3H mouse.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Potential risk of inadvertent germ-line gene transmission statement from the American Society of Gene Therapy to the NIH Recombinant DNA Advisory Committee, March 12, 1999.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Adeno-associated virus and development of cervical neoplasia

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Evaluation of the possible protective role of adeno-associated virus type 2 infection in HPV-associated premalignant disease of the cervix

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Recombinant adeno-associated virus for muscle directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Additional transduction events after subretinal readministration of recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Factors influencing cross-presentation of non-self antigens expressed from recombinant adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Major histocompatibility complex class I-restricted cross-presentation is biased towards high dose antigens and those released during cellular destruction

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Induction of tolerance to human factor VIII in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Many tumors induced by the mouse mammary tumor virus contain a provirus integrated in the same region of the host genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Activation of a cellular onc gene by promoter insertion in ALV-induced lymphoid leukosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Insertion vectors for gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Insertional mutagenesis of the adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Glucose-responsive gene delivery in pancreatic Islet cells via recombinant adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Development of formulations that enhance physical stability of viral vectors for gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Standard heparin, low molecular weight heparin, low molecular weight heparinoid, and recombinant hirudin differ in their ability to inhibit transduction by recombinant adeno-associated virus type 2 vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Virus-mediated killing of cells that lack p53 activity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

Dilute (d) coat colour mutation of DBA/2J mice is associated with the site of integration of an ecotropic MuLV genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14740338.1.1.79

21 January 2018

Identifiers

DOI

10.1517/14740338.1.1.79

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7 August 2017

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