Gene therapy returns to centre stage (Q38607706)

4 September 2017

title

Gene therapy returns to centre stage (English)

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4 September 2017

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1

Luigi Naldini

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language of work or name

English

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publication date

1 October 2015

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4 September 2017

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4 September 2017

volume

526

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4 September 2017

issue

7573

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4 September 2017

page(s)

351-360

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Targeted gene therapy and cell reprogramming in Fanconi anemia

4 September 2017

cites work

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12 December 2020

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

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12 December 2020

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

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12 December 2020

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

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12 December 2020

Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency

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12 December 2020

Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort

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12 December 2020

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

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12 December 2020

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.

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12 December 2020

CRISPR germline engineering--the community speaks

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12 December 2020

Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV

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12 December 2020

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

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12 December 2020

Improving the safety of cell therapy with the TK-suicide gene

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12 December 2020

Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

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12 December 2020

Going viral with cancer immunotherapy

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12 December 2020

Chimeric antigen receptor T cells for sustained remissions in leukemia

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12 December 2020

Hematopoiesis: a human perspective

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12 December 2020

In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells

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12 December 2020

T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial

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12 December 2020

Cancer immunology. Mutational landscape determines sensitivity to PD-1 blockade in non-small cell lung cancer

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12 December 2020

Genome editing. The new frontier of genome engineering with CRISPR-Cas9

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12 December 2020

$1-million price tag set for Glybera gene therapy

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12 December 2020

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

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12 December 2020

Regulatory evaluation of Glybera in Europe - two committees, one mission

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12 December 2020

In vivo genome editing using Staphylococcus aureus Cas9

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12 December 2020

Overcoming preexisting humoral immunity to AAV using capsid decoys

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12 December 2020

Treatment of metastatic melanoma with autologous CD4+ T cells against NY-ESO-1

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12 December 2020

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

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12 December 2020

Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

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12 December 2020

A Monovalent Chimpanzee Adenovirus Ebola Vaccine Boosted with MVA

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12 December 2020

Vectored immunoprophylaxis protects humanized mice from mucosal HIV transmission

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12 December 2020

An unbiased genome-wide analysis of zinc-finger nuclease specificity

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12 December 2020

Prime-boost vaccination with chimpanzee adenovirus and modified vaccinia Ankara encoding TRAP provides partial protection against Plasmodium falciparum infection in Kenyan adults

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12 December 2020

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

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12 December 2020

Long-term effects of retinal gene therapy in childhood blindness

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12 December 2020

Primary immunodeficiencies: 2009 update

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12 December 2020

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

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12 December 2020

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR.

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12 December 2020

An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level.

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12 December 2020

Fanconi anemia gene editing by the CRISPR/Cas9 system

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12 December 2020

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

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12 December 2020

The future of immune checkpoint therapy

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12 December 2020

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

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12 December 2020

Long-term effect of gene therapy on Leber's congenital amaurosis

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12 December 2020

A pilot trial using lymphocytes genetically engineered with an NY-ESO-1-reactive T-cell receptor: long-term follow-up and correlates with response

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12 December 2020

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

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12 December 2020

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

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12 December 2020

CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia

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12 December 2020

Adoptive cellular therapy: a race to the finish line.

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12 December 2020

Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases

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12 December 2020

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

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12 December 2020

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

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12 December 2020

Therapeutic genome editing: prospects and challenges

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12 December 2020

Modern haemophilia care

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12 December 2020

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

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12 December 2020

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

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12 December 2020

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

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12 December 2020

The special case of gene therapy pricing

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12 December 2020

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

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12 December 2020

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

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12 December 2020

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

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12 December 2020

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

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12 December 2020

Adeno-associated virus vectorology, manufacturing, and clinical applications

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12 December 2020

Physiological promoters reduce the genotoxic risk of integrating gene vectors

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12 December 2020

Hematopoietic stem-cell transplantation

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12 December 2020

Ex vivo gene transfer and correction for cell-based therapies

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12 December 2020

Current status of haemophilia gene therapy

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12 December 2020

Improvement and decline in vision with gene therapy in childhood blindness.

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12 December 2020

X-linked adrenoleukodystrophy: clinical, metabolic, genetic and pathophysiological aspects.

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12 December 2020

Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters.

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12 December 2020

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

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12 December 2020

Engineering adeno-associated viruses for clinical gene therapy

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12 December 2020

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

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12 December 2020

Predicting immunogenic tumour mutations by combining mass spectrometry and exome sequencing.

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12 December 2020

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

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12 December 2020

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

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12 December 2020

Biotechnology. A prudent path forward for genomic engineering and germline gene modification

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12 December 2020

Adoptive immunotherapy for cancer or viruses

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12 December 2020

Metachromatic leukodystrophy--an update

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12 December 2020

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

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12 December 2020

Long-term follow-up after gene therapy for canavan disease.

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12 December 2020

Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial

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12 December 2020

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.

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12 December 2020

Concise review: managing genotoxicity in the therapeutic modification of stem cells

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12 December 2020

Progress and prospects: immune responses to viral vectors

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12 December 2020

In vivo genome editing restores haemostasis in a mouse model of haemophilia

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12 December 2020

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity

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12 December 2020

Adoptive cell transfer as personalized immunotherapy for human cancer

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12 December 2020

Cancer regression and autoimmunity in patients after clonal repopulation with antitumor lymphocytes

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12 December 2020

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

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12 December 2020

Gene therapy outpaces haplo for SCID-X1.

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12 December 2020

Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs.

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12 December 2020

The road to purified hematopoietic stem cell transplants is paved with antibodies.

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12 December 2020

Chemotherapy-refractory diffuse large B-cell lymphoma and indolent B-cell malignancies can be effectively treated with autologous T cells expressing an anti-CD19 chimeric antigen receptor

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12 December 2020

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

Memory T cells officially join the stem cell club.

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12 December 2020

Checkpoint blockade cancer immunotherapy targets tumour-specific mutant antigens

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12 December 2020

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

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12 December 2020

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

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12 December 2020

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

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12 December 2020

The AAV vector toolkit: poised at the clinical crossroads

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12 December 2020

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

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12 December 2020

Neoantigens in cancer immunotherapy.

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12 December 2020

Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector

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12 December 2020

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

1 reference

12 December 2020

Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma.

1 reference

12 December 2020

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

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12 December 2020

New viruses for cancer therapy: meeting clinical needs

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12 December 2020

Site-specific integration and tailoring of cassette design for sustainable gene transfer.

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12 December 2020

Repair of strand breaks by homologous recombination

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12 December 2020

Targeted genome editing in human repopulating haematopoietic stem cells

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12 December 2020

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

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12 December 2020

Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen

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12 December 2020

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

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12 December 2020

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

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12 December 2020

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

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12 December 2020

Efficacy of gene therapy for X-linked severe combined immunodeficiency

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12 December 2020

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.

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12 December 2020

Assessing the potential for AAV vector genotoxicity in a murine model

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12 December 2020

20 years of gene therapy for SCID.

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https://api.crossref.org/works/10.1038%2FNATURE15818

7 January 2021

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

1 reference

https://api.crossref.org/works/10.1038%2FNATURE15818

7 January 2021

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

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https://api.crossref.org/works/10.1038%2FNATURE15818

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNATURE15818

7 January 2021

Identifiers

DOI

10.1038/NATURE15818

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Dimensions Publication ID

4 September 2017

1051703844

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Fatcat ID

release_gzs6te7arzfd7dwuwgjztx3ova

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Fatcat

https://api.fatcat.wiki/v0/release/gzs6te7arzfd7dwuwgjztx3ova

24 November 2022

mapped directly with Wikidata item

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