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Harnessing a novel CRISPR nuclease for programmable counterselection in human cells

Descrizione del progetto

Progressi delle tecniche di editing del genoma

Le tecnologie CRISPR, che consentono di modificare con precisione il DNA negli organismi viventi, hanno rivoluzionato l’editing del genoma. Si basano sui meccanismi di difesa naturali dei batteri contro i virus. Nonostante il loro potenziale, le basse frequenze di editing pongono delle sfide, rendendo necessario uno screening approfondito. Il progetto CRISPR-SELECT, finanziato dal Consiglio europeo della ricerca, propone una svolta nella selezione delle cellule modificate eliminando quelle non modificate. Questo approccio promette di aumentare in modo significativo le frequenze di editing, alleggerendo l’onere dello screening nelle varie applicazioni di editing genomico. La proposta delinea esperimenti dimostrativi in cellule umane, esplorando le potenziali applicazioni di questa innovazione tecnologica.

Obiettivo

CRISPR technologies have revolutionized genome editing in medicine, agriculture, biotechnology, and life-sciences research with their ability to generate virtually any DNA edit at any genomic site in any organism. However, editing frequencies can be restrictively low, requiring extensive screening to identify cells harboring desired edits. What remains elusive is a way to greatly boost the frequency of editing. While characterizing CRISPR-Cas systems, bacterial defense systems and the source of CRISPR technologies, we discovered a new mechanism that could kill unedited cells yet spare edited cells, regardless of the type of gene edit. This versatile and sequence-specific approach can be described as programmable counter-selection, as undesired cells are targeted for removal, and those cells can be targeted in a programmable way. If proven, this capability could radically boost the effective editing frequencies by removing unedited cells regardless of the underlying edit, providing much-needed relief to the burden of screening imposed on diverse genome-editing applications. Here, we propose to perform proof-of-concept experiments demonstrating this capability in human cells while exploring which editing applications would best benefit from this capability. The associated tasks build on my extensive work at the interface of CRISPR biology and technologies and leverages his numerous academic and industrial contacts. I ultimately aim to translate a novel biological insight from my group’s ERC Consolidator project into an innovative foundational technology, with a clear path toward its broad use in genome editing.

Parole chiave

Istituzione ospitante

HELMHOLTZ-ZENTRUM FUR INFEKTIONSFORSCHUNG GMBH
Contribution nette de l'UE
€ 150 000,00
Indirizzo
INHOFFENSTRASSE 7
38124 Braunschweig
Germania

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Regione
Niedersachsen Braunschweig Braunschweig, Kreisfreie Stadt
Tipo di attività
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Costo totale
Nessun dato

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