Gene therapy: trials and tribulations (Q34186665)

31 July 2017

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title

Gene therapy: trials and tribulations (English)

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31 July 2017

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Somia N

1

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31 July 2017

Verma IM

series ordinal

2

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31 July 2017

publication date

1 November 2000

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31 July 2017

published in

Nature Reviews Genetics

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31 July 2017

volume

1

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31 July 2017

issue

2

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31 July 2017

page(s)

91-99

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https://scigraph.springernature.com/pub.10.1038/35038533

31 July 2017

exact match

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cites work

Nonviral gene therapy: promises and challenges

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A triploblast origin for Myxozoa?

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The basic science of gene therapy

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Human gene therapy comes of age.

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A safe packaging line for gene transfer: separating viral genes on two different plasmids

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Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges

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Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells

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Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells

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An alternative approach to somatic cell gene therapy

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Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes

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Human immunodeficiency virus infection of cells arrested in the cell cycle

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A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells

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HIV-1: fifteen proteins and an RNA

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In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

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Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

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Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

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HIV-1 genome nuclear import is mediated by a central DNA flap

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Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

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Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

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Development of a self-inactivating lentivirus vector.

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Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus

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Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors

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Stable gene transfer to the nervous system using a non-primate lentiviral vector

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Site-specific integration by adeno-associated virus

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High-fidelity correction of mutations at multiple chromosomal positions by adeno-associated virus vectors.

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Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions

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Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2.

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

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Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy

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Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors

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Advances in adenoviral vectors: from genetic engineering to their biology

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7 January 2021

A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase

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A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal

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Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons

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7 January 2021

Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression

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7 January 2021

Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: implications for gene therapy.

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7 January 2021

Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors

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7 January 2021

Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle

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Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes

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Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.

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Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors.

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Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

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T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

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T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

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Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

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Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

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7 January 2021

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

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7 January 2021

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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7 January 2021

Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

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7 January 2021

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

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7 January 2021

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7 January 2021

Retroviral vectors preloaded with a viral receptor-ligand bridge protein are targeted to specific cell types

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7 January 2021

Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody

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7 January 2021

Tight control of gene expression in mammalian cells by tetracycline-responsive promoters

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7 January 2021

Exploring the sequence space for tetracycline-dependent transcriptional activators: novel mutations yield expanded range and sensitivity

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7 January 2021

Lentiviral vectors: regulated gene expression

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7 January 2021

A regulatory system for use in gene transfer

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7 January 2021

Ecdysone-inducible gene expression in mammalian cells and transgenic mice.

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7 January 2021

High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor

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7 January 2021

Controlling signal transduction with synthetic ligands

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7 January 2021

Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer.

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7 January 2021

Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector

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7 January 2021

Identifiers

DOI

10.1038/35038533

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Dimensions Publication ID

31 July 2017

1012109533

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