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The fusion of a programmable transcription-activator-like effector (TALE) protein with a nickase, in conjunction with a deaminase, enables efficient and strand-selective DNA base editing. This approach has the potential to advance our understanding and treatment of diseases associated with mutations in the mitochondrial or nuclear genome.
Minigraph-Cactus, a method to efficiently combine multiple reference genome assemblies into a pangenome reference graph, can be used to improve accuracy of read mapping and variant calling compared with a single reference in downstream applications.
By linking transgene expression with that of a housekeeping gene, SLEEK (selection by essential-gene exon knock-in) enables efficient knock-in of complex cargos in a variety of clinically relevant cell types. Using SLEEK, we were able to substantially improve the tumor suppression ability and in vivo persistence of a cell therapy.
Small molecules that target messenger RNA have therapeutic potential, but the field still lacks an unqualified success. Companies differ on how to move the resurgent field forward.