Titolo progetto: Developing chromosome transplant technologies towards universal cell therapy

Programma di finanziamento: Bando giovani ricercatori MUR.

PNRR - Missione 4 “Istruzione e Ricerca” - Componente 2 “Dalla Ricerca all'Impresa” - Investimento 1.2 “Finanziamento di progetti presentati da giovani ricercatori”

Responsabile scientifico: dott. Gianluca Petris

Ruolo del DMED: Coordinatore

Descrizione generale:

Rare genetic disease affect 3-6% of the human population. In late years, some gene and cell therapies showed efficacy and received therapeutic approval for the treatment of certain rare diseases. Unfortunately, the scarcity of patients for each specific genetic mutation requiring a correction, or for each affected gene involved, in each rare genetic disease resulted in the development of personalized, but extremely expensive and unsustainable, medicinal products. In several cases the clinical application of effective drugs has been disabled due to industrial economic reasons. Moreover, classic genome editing, gene and cell therapy approaches are unable, or struggle, to address large and multigene genetic defects.

Intact chromosome transplants can offer the opportunity for developing universal genome engineering strategies, enabling mutation and gene-agnostic chromosome-scale genetic corrections for universal cell therapies. This will enable to cluster candidate patients in chromosome and affected organ-specific manner.

I have recently developed methods for whole chromosome transplants, capable of transferring any human chromosome between stem and somatic cells, even across species. I have designed the methodologies to minimise genome toxicity, achieving intact chromosome transplant without unexpected genome damage. These results encourage pursuing chromosome transplant for disease modelling and therapy.

I will validate intact chromosome transplant technologies in candidate cells (e.g. hiPSC, liver organoids) suitable for cell therapies in highly regenerative organs (e.g. liver, hematopoietic system).

The chromosome-scale genome engineering strategy, demonstrated in preclinical settings by this project, will pave the way for developing novel, mutation-agnostic, broadly applicable cell therapeutics.

Date inizio e fine progetto:  20/05/2025 – 19/05/2028

Budget totale del progetto: € 299.950,50

Finanziato dall’Unione europea- Next Generation EU, Missione 4 Componente 2 CUP G23C25000630006.