Hematopoietic Stem Cell (HSC) Targeted Gene Editing and Methods of Use Thereof
AssigneesThe Trustees of the University of Pennsylvania, The Children's Hospital of Philadelphia
InventorsHamideh Parhiz, Tyler Papp, Stefano Rivella, Laura Breda, Michael Triebwasser
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Abstract
The present invention relates to CD117 targeted LNP molecules for delivery of gene editing molecules to hematopoietic stem cells (HSCs) and methods of use thereof for base editing of genomic DNA for the treatment of a disease or disorder.
Claims16
A composition for targeted delivery of gene editing agents to a target hematopoietic stem cell (HSC), the composition comprising a first delivery vehicle comprising an mRNA molecule encoding a Cas9 base editor and a second delivery vehicle comprising a short guide RNA (sgRNA), wherein each of the first and second delivery vehicle comprises a CD117 targeting moiety.
Dependencies are inferred from the claim text.