… Koller and CF Bennett, manuscript in preparation). Studies of these cells demonstrate that,
in contrast to most cultured cells (106, 181), these hepatocellular carcinoma cells accumulate …

Pharmacokinetic properties of oligonucleotides are largely driven by chemistry of the backbone
and thus are sequence independent within a chemical class. Tissue bioavailability (% of …

RNA-targeted therapies represent a platform for drug discovery involving chemically modified
oligonucleotides, a wide range of cellular RNAs, and a novel target-binding motif, Watson-…

MicroRNAs (miRNAs) circulate in the bloodstream in a highly stable, extracellular form and
are being developed as blood-based biomarkers for cancer and other diseases. However, …

Current understanding of microRNA (miRNA) biology is limited, and antisense oligonucleotide
(ASO) inhibition of miRNAs is a powerful technique for their functionalization. To uncover …

Alternative splicing (AS) of pre-mRNA is utilized by higher eukaryotes to achieve increased
transcriptome and proteomic complexity. The serine/arginine (SR) splicing factors regulate …

Background Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that
is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an …

We used cross-linking and immunoprecipitation coupled with high-throughput sequencing
to identify binding sites in 6,304 genes as the brain RNA targets for TDP-43, an RNA binding …

MicroRNAs (miRNAs) are endogenously expressed 20-24 nucleotide RNAs thought to
repress protein translation through binding to a target mRNA (1-3). Only a few of the more than …

Background Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger
RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the …