National Cancer Institute 5R01CA283364-03

Synthetic introns for selective targeting of RNA splicing factor-mutant leukemia

The project funded by the National Cancer Institute focuses on developing synthetic intron-based precision therapeutics to selectively target and eliminate cancer cells with RNA splicing factor mutations, addressing critical needs in treating myeloid neoplasms such as MDS, AML, and CMML.

Description

This project aims to develop synthetic intron-based therapeutics for myeloid neoplasms, including MDS, AML, and CMML, by creating precision therapeutics that selectively kill cells with spliceosomal mutations using synthetic intron technology. The work involves dissecting molecular mechanisms, developing synthetic introns for therapeutic protein expression, and optimizing in vivo delivery and immunostimulatory therapies. The significance is to develop a technology enabling mechanistic studies of spliceosomal mutations and providing targeted therapy for cancers with high unmet medical needs.

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