Splicing deregulation detected and targeted in type of childhood leukemia ucsandiego CellReports CellRepMed
"Our hypothesis was that the drivers were not genomic alterations, but rather occurred within transcriptomic and epitranscriptomic processes."
The transcriptome describes all of the RNA expressed by genes in an organism, such as a person. RNA is used to convey the genetic information needed to make proteins and perform or regulate cellular functions. Transcriptomics is used to learn how genes are turned on and off in different cells and how that may be linked to certain diseases. The epitranscriptome refers to all of the modifications to RNA that occur within a cell.
In their study, Jamieson and colleagues at UC San Diego and in The Netherlands pinpointed a deregulation in splicing—part of the process of decoding gene instructions into proteins—that causes a therapeutic vulnerability to a small molecule splicing inhibitor called Rebecsinib.
"Our findings suggest a new approach to detecting and targeting therapy-resistant leukemia stem cells in pediatric AML and other that become recalcitrant to treatment as a consequence of this splicing deregulation," Jamieson said.An estimated 54,000 children and adolescents in the United States are living with or in remission from blood cancers, with leukemia the most common, accounting for more than one-quarter of cases.
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