Gene therapy for primary immunodeficiencies: current status and future prospects (Q38213501)

31 August 2017

Gene therapy for primary immunodeficiencies: current status and future prospects (English)

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31 August 2017

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Waseem Qasim

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Andrew R Gennery

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2

object named as

Andrew R Gennery

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31 August 2017

1 June 2014

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31 August 2017

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31 August 2017

74

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963-969

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31 August 2017

9

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https://scigraph.springernature.com/pub.10.1007/s40265-014-0223-7

31 August 2017

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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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Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

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Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor.

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Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

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

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Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

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Efficacy of gene therapy for X-linked severe combined immunodeficiency

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

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

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Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

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Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

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Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

21 January 2018

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

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

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Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000.

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Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft

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Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

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Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice

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Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors

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Highly efficient endogenous human gene correction using designed zinc-finger nucleases

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Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy

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Targeted genome editing across species using ZFNs and TALENs

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Genome engineering using the CRISPR-Cas9 system

21 January 2018

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

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Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients

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Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity

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Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

21 January 2018

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Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years

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Unresolved issues in hematopoietic stem cell transplantation for severe combined immunodeficiency: need for safer conditioning and reduced late effects

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SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID.

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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

Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening

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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

Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency

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12 December 2020

10.1007/S40265-014-0223-7

Identifiers

DOI

1 reference

PubMed ID

24848753

Dimensions Publication ID

31 August 2017

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1 reference

PubMed ID

24848753