TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage
Eduardo Seclen,
Jessica C. Jang,
Aminah O. Lawal
et al.
Abstract:Gene therapy in hematopoietic stem and progenitor cells (HSPCs) shows great potential for the treatment of inborn metabolic diseases. Typical HSPC gene therapy approaches rely on constitutive promoters to express a therapeutic transgene, which is associated with multiple disadvantages. Here, we propose a novel promoter-less intronic gene editing approach that triggers transgene expression only after cellular differentiation into the myeloid lineage. We integrated a splicing-competent eGFP cassette into the fir… Show more
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