Article

TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage

2024-03-05

Abstract excerpt

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 diffe...

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Literature Corpus work: db575135-9706-5b77-b160-edc4fe4b53ad
DOI: 10.1101/2024.03.05.583596

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