RNA-loaded nanoparticles for the treatment of hematological cancers

• NPs play a crucial role in RNA-based hematological tumor therapies. • RNA-NPs have the ability to modify genetic and epigenetic alterations or the tumor microenvironment. • In vivo CAR-T cell generation is achievable through RNA-loaded NPs. • Pediatric patients with blood cancers may experience benefits from RNA-loaded NPs. • NPs significantly improve the delivery and efficacy of RNA therapy in preclinical studies. Hematological cancers encompass a diverse group of malignancies affecting the blood, bone marrow, lymph nodes, and spleen. These disorders present unique challenges due to their complex etiology and varied clinical manifestations. Despite significant advancements in understanding and treating hematological malignancies, innovative therapeutic approaches are continually sought to enhance patient outcomes. This review highlights the application of RNA nanoparticles (RNA-NPs) in the treatment of hematological cancers. We delve into detailed discussions on in vitro and preclinical studies involving RNA-NPs for adult patients, as well as the application of RNA-NPs in pediatric hematological cancer. The review also addresses ongoing clinical trials involving RNA-NPs and explores the emerging field of CAR-T therapy engineered by RNA-NPs. Finally, we discuss the challenges still faced in translating RNA-NP research to clinics.