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TACKLING FUNCTIONAL MATURATION FOR TRANSPLANTABLE HEMATOPOIETIC STEM CELL GENERATION

Project description

Advancing the therapeutic potential of haematopoietic stem cells

Transplantation of hematopoietic stem cells (HSC) has traditionally been employed for the treatment of haematological diseases. Recent gene therapy applications using HSCs have spurred great interest in these cells beyond haematology. However, challenges like donor matching and cell quantity limit efficacy and the widespread application of HSC transplantation. Funded by the European Research Council, the FUN-HSC project aims to create a reliable source of HSCs from induced pluripotent stem cells. Researchers will investigate core maturation pathways and gene expression regulation, providing important insight into HSC biology and an engraftable source for advanced therapeutic applications.

Objective

Hematopoietic stem cells (HSC) provide a lifelong supply of blood and immune cells. They are a life-saving therapy for leukemia and have recently found broadened applications to gene and cell therapy, against other cancers, genetic, autoimmune, and infectious diseases. Nonetheless, practical limitations, such as donor-matching or insufficient cell number render these therapies unfeasible or unsuccessful for many patients. Attempts to overcome these limitations have been unable to create a reliable source of HSC for patients: while we can generate human HSC from pluripotent stem cells (PSC), they resemble early development stages, lacking the functional maturation hallmarks. FUN-HSC aims at identifying core and actionable maturation pathways for HSCs and at defining the contribution of coordinated gene expression by chromatin regulation, using a combination of functional and genetic screening technologies and high-throughput readouts in vitro and in vivo. HSC maturation occurs in the liver during fetal development and confers functional multilineage hematopoiesis ability throughout adulthood. Using single cell omics analysis of human embryonic and fetal tissues, we describe multiple gene programs changing during developmental HSC maturation. This study will lead to candidate cell-intrinsic and niche-dependent instructions, which will be dissected in the context of the fetal liver, by analyzing the cell compartments and their interactions and by combining novel organoid technologies with mouse xenografts to model the fetal liver environment. Ultimately, we aim to identify a strategy that mimics these pathways in vitro to bring the maturation stage of the PSC-derived HSC, to a robustly engraftable and clinically valuable HSC source. Harnessing new knowledge in human HSC biology, cutting-edge genetics and tissue technologies, FUN-HSC will provide great advances in the mission of making and maintaining HSCs in vitro, enabling their full therapeutic potential.

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Topic(s)

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HORIZON-ERC - HORIZON ERC Grants

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Call for proposal

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(opens in new window) ERC-2022-COG

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Host institution

FUNDACIO INSTITUT DE RECERCA CONTRA LA LEUCEMIA JOSEP CARRERAS
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 2 065 784,00
Address
CARRETERA DE CAN RUTI CAMI DE LES ESCOLES S/N
08916 Badalona
Spain

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Region
Este Cataluña Barcelona
Activity type
Research Organisations
Links
Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

€ 2 065 784,00

Beneficiaries (2)

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