Human gene-engineered calreticulin mutant stem cells recapitulate mpn hallmarks and identify targetable vulnerabilities

HIGHLIGHTS

• who: Johannes Fou00dfelteder from the (UNIVERSITY) have published the article: Human gene-engineered calreticulin mutant stem cells recapitulate MPN hallmarks and identify targetable vulnerabilities, in the Journal: (JOURNAL) of 22/02/2023
• what: The authors demonstrate a genome engineering system with targeted integration of type 1 and type 2 CALR mutations into healthy HSPCs at their natural genomic loci, preserving endogenous expression . Since CALR mutations mainly occur heterozygously , the authors aimed to guarantee this genotype by simultaneously integrating MUT and WT cDNAs coupled with distinct fluorescent reporters on individual alleles.

SUMMARY

 



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