Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 May 2018

Hiramoto Takafumi, Li Li B, Funk Sarah E, Hirata Roli K, Russell David W

Abstract excerpt

X-linked severe combined immunodeficiency (X-SCID) has been successfully treated by hematopoietic stem cell (HSC) transduction with retroviral vectors expressing the interleukin-2 receptor subunit gamma gene (IL2RG), but several patients developed malignancies due to vector integration near cellular oncogenes. This adverse side effect could in principle be avoided by accurate IL2RG gene editing with a vector that...

Topics

Alleles
Animals
Bone Marrow Cells
Dependovirus
Gene Editing
Gene Order
Genetic Therapy
Genetic Vectors
Hematopoietic Stem Cells
Humans
Immunotherapy, Adoptive

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Interleukin Receptor Common gamma Subunit

Mice

Receptors, Antigen, T-Cell

T-Lymphocytes

X-Linked Combined Immunodeficiency Diseases

Source

Open in PubMed Open full text

Identifiers

PMID: 29606506
DOI: 10.1016/j.ymthe.2018.02.028
PMCID: PMC5993949

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

Community interpretation

Discuss the evidence

Ask a question, add context or challenge an interpretation. Community activity is separate from scientific evidence strength.

0

discussions

0

replies

Citations encouraged

Claims remain attributable.

No discussion yet

Be the first to add a careful, cited interpretation.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice.PMID 29606506Read ↗

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined ImmunodeficiencySemantic proximity Read ↗

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cellsSemantic proximity Read ↗

Targeted genome editing in human repopulating haematopoietic stem cells.Semantic proximity Read ↗

Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus.Semantic proximity Read ↗

Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.Semantic proximity Read ↗

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.Semantic proximity Read ↗

Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem CellsSemantic proximity Read ↗

Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency diseaseSemantic proximity Read ↗

CRISPR-Cas9 engineering of the RAG2 locus via complete coding sequence replacement for therapeutic applicationsSemantic proximity Read ↗

Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime EditingSemantic proximity Read ↗

Clinical Applications of Gene Therapy for Primary ImmunodeficienciesSemantic proximity Read ↗

Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiencySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.