ArticleMolecular therapy : the journal of the American Society of Gene Therapy2011
Development of B-lineage predominant lentiviral vectors for use in genetic therapies for B cell disorders.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed, 34 citations in OpenAlex.
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- A comparison of DNA repair pathways to achieve a site-specific gene modification of the Bruton's tyrosine kinase gene.Molecular therapy. Nucleic acids · 2022Article
- An instructive role for Interleukin-7 receptor α in the development of human B-cell precursor leukemia.Nature communications · 2022Article
- Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia.The CRISPR journal · 2021Article
- Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector.Molecular therapy. Methods & clinical development · 2021Article
- Gene Therapy for Primary Immunodeficiency.HemaSphere · 2021Review
- Generation of a cost-effective cell line for support of high-throughput isolation of primary human B cells and monoclonal neutralizing antibodies.Journal of immunological methods · 2021Article
- Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.Molecular therapy. Methods & clinical development · 2017Article
- The BANK1 SLE-risk variants are associated with alterations in peripheral B cell signaling and development in humans.Clinical immunology (Orlando, Fla.) · 2016Article
- Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Progressive engineering of a homing endonuclease genome editing reagent for the murine X-linked immunodeficiency locus.Nucleic acids research · 2014Article
- Epstein-Barr virus utilizes Ikaros in regulating its latent-lytic switch in B cells.Journal of virology · 2014Article
- megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.Nucleic acids research · 2014Article
- Kinase-independent feedback of the TAK1/TAB1 complex on BCL10 turnover and NF-κB activation.Molecular and cellular biology · 2013Article
- Coupling endonucleases with DNA end-processing enzymes to drive gene disruption.Nature methods · 2012Article
- Engineering domain fusion chimeras from I-OnuI family LAGLIDADG homing endonucleases.Nucleic acids research · 2012Article
- Article
- Engineering humanized mice for improved hematopoietic reconstitution.Cellular & molecular immunology · 2012Review
- Developmentally regulated expression of MEF2C limits the response to BCR engagement in transitional B cells.European journal of immunology · 2012Article
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 2 countries.
Funding
Abstract
Sustained, targeted, high-level transgene expression in primary B lymphocytes may be useful for gene therapy in B cell disorders. We developed several candidate B-lineage predominant self-inactivating lentiviral vectors (LV) containing alternative enhancer/promoter elements including: the immunoglobulin β (Igβ) (B29) promoter combined with the immunoglobulin µ enhancer (EµB29); and the endogenous BTK promoter with or without Eµ (EµBtkp or Btkp). LV-driven enhanced green fluorescent protein (eGFP) reporter expression was evaluated in cell lines and primary cells derived from human or murine hematopoietic stem cells (HSC). In murine primary cells, EµB29 and EµBtkp LV-mediated high-level expression in immature and mature B cells compared with all other lineages. Expression increased with B cell maturation and was maintained in peripheral subsets. Expression in T and myeloid cells was much lower in percentage and intensity. Similarly, both EµB29 and EµBtkp LV exhibited high-level activity in human primary B cells. In contrast to EµB29, Btkp and EµBtkp LV also exhibited modest activity in myeloid cells, consistent with the expression profile of endogenous Bruton's tyrosine kinase (Btk). Notably, EµB29 and EµBtkp activity was superior in all expression models to an alternative, B-lineage targeted vector containing the EµS.CD19 enhancer/promoter. In summary, EµB29 and EµBtkp LV comprise efficient delivery platforms for gene expression in B-lineage cells.
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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.