Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial (Q44328165)

2 December 2017

Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial (English)

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2 December 2017

Joerg Schuettrumpf

1

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2 December 2017

Jian-Hua Liu

2

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2 December 2017

Linda B Couto

3

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2 December 2017

Kathakaly Addya

4

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2 December 2017

Debra G B Leonard

5

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2 December 2017

Zhu Zhen

6

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2 December 2017

Jürg Sommer

7

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2 December 2017

Valder R Arruda

8

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2 December 2017

2 May 2006

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2 December 2017

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2 December 2017

13

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2 December 2017

1064-1073

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2 December 2017

6

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Gene therapy clinical trials worldwide 1989-2004-an overview.

2 December 2017

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Germline alteration by gene therapy: assessing and reducing the risks

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Male germ-line cells are at risk following direct-injection retroviral-mediated gene transfer in utero

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Detection and quantification of HIV-1 in semen: identification of a subpopulation of men at high potential risk of viral sexual transmission

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Viruses in the mammalian male genital tract and their effects on the reproductive system

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

No evidence for germ-line transmission following prenatal and early postnatal AAV-mediated gene delivery.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Direct exposure of mouse spermatozoa to very high concentrations of a serotype-2 adeno-associated virus gene therapy vector fails to lead to germ cell transduction

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

An estimated frequency of endogenous insertional mutations in humans

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Adeno-associated virus vectors can be efficiently produced without helper virus

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

Generation and characterization of chimeric recombinant AAV vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.03.002

10.1016/J.YMTHE.2006.03.002

7 January 2021

Identifiers

DOI

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2 December 2017

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