ArticleMolecular therapy. Methods & clinical development2015
Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy.
Article in Molecular therapy. Methods & clinical development, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
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The trial behind it
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Who cites it
18 citing papers in PubMed, 27 citations in OpenAlex.
- Genome editing with the HDR-enhancing DNA-PKcs inhibitor AZD7648 causes large-scale genomic alterations.Nature biotechnology · 2025Article
- Challenges in lentiviral vector production: retro-transduction of producer cell lines.Frontiers in bioengineering and biotechnology · 2025Article
- An optimized protocol for quality control of gene therapy vectors using nanopore direct RNA sequencing.Genome research · 2024Article
- Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning.Molecular therapy. Methods & clinical development · 2023Article
- Lentiviral Vector Bioprocessing.Viruses · 2021Review
- Viral vector platforms within the gene therapy landscape.Signal transduction and targeted therapy · 2021Review
- Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media.Molecular therapy. Methods & clinical development · 2020Article
- High level of fetal-globin reactivation by designed transcriptional activator-like effector.Blood advances · 2020Article
- Lentivector Producer Cell Lines with Stably Expressed Vesiculovirus Envelopes.Molecular therapy. Methods & clinical development · 2018Article
- Staurosporine Increases Lentiviral Vector Transduction Efficiency of Human Hematopoietic Stem and Progenitor Cells.Molecular therapy. Methods & clinical development · 2018Article
- Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome.Journal of clinical immunology · 2018Review
- Scalable Lentiviral Vector Production Using Stable HEK293SF Producer Cell Lines.Human gene therapy methods · 2017Article
- Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.Molecular therapy. Methods & clinical development · 2017Article
- Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Development of Third-generation Cocal Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T-cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- RD-MolPack technology for the constitutive production of self-inactivating lentiviral vectors pseudotyped with the nontoxic RD114-TR envelope.Molecular therapy. Methods & clinical development · 2016Article
- Production of lentiviral vectors.Molecular therapy. Methods & clinical development · 2016Review
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
We have developed a producer cell line that generates lentiviral vector particles of high titer. The vector encodes the Wiskott-Aldrich syndrome (WAS) protein. An insulator element has been added to the long terminal repeats of the integrated vector to limit proto-oncogene activation. The vector provides high-level, stable expression of WAS protein in transduced murine and human hematopoietic cells. We have also developed a monoclonal antibody specific for intracellular WAS protein. This antibody has been used to monitor expression in blood and bone marrow cells after transfer into lineage negative bone marrow cells from WAS mice and in a WAS negative human B-cell line. Persistent expression of the transgene has been observed in transduced murine cells 12-20 weeks following transplantation. The producer cell line and the specific monoclonal antibody will facilitate the development of a clinical protocol for gene transfer into WAS protein deficient stem cells.
Identifiers
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.