Article

Targeted gene addition at the CX3CR1 locus of human HSPCs improves their ability to repopulate the myeloid CNS compartment and drives lineage specific and robust transgene expression in their CNS progeny

2023-07-12

Abstract excerpt

<title>Abstract</title> <p>Transplantation of engineered hematopoietic stem and progenitor cells (HSPCs) showed curative potential in patients affected by neurometabolic diseases treated in early stage. Favoring the engraftment and maturation of the engineered HSPCs in the central nervous system (CNS) could allow enhancing further the therapeutic potential of this approach. To this goal, we propose a gene additio...

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Literature Corpus work: e0525019-4d6b-5052-a16f-0990f91e464c
DOI: 10.21203/rs.3.rs-3057675/v1

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Targeted gene addition at the CX3CR1 locus of human HSPCs improves their ability to repopulate the myeloid CNS compartment and drives lineage specific and robust transgene expression in their CNS progenyDOI 10.21203/rs.3.rs-3057675/v1Read

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