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Transcriptome-wide effects of gene knockouts are screened at the single-cell level by combining high-throughput, single-cell RNA-seq with lentiviral CRISPR libraries.
Refinements in the chemistries employed in oligonucleotide therapeutics have galvanized clinical progress. The complex interplay between chemical modifications and integration into sequence architecture is discussed in the context of antisense and small-interfering RNA drugs.