Adrian R. Krainer

This study identifies BCAS2 as a critical regulator of PDAC progression by promoting alternative splicing of weak 5' splice sites, specifically enhancing SOS1 exon 21 inclusion and RAC-signaling activation.

This study identifies splicing dysregulation in SF-mutated cancers and demonstrates that antisense oligonucleotides can correct aberrant splicing and restore tumor suppressor function.

This study demonstrates that exon-skipping antisense oligonucleotides targeting H3.3K27M-altered DMG reduce proliferation and extend survival in a mouse model.

The paper identifies a MYC-USP39-SRSF1 regulatory axis in PDAC that stabilizes SRSF1 and promotes tumor progression, suggesting USP39 as a therapeutic target.

This study shows CDK11 is critical for cancer cell survival and identifies 1p36 deletions as a biomarker for CDK11 inhibitor sensitivity, but on-target toxicity limits therapeutic efficacy.

This study identifies an SRSF1-AURKA-MYC oncogenic circuit in pancreatic cancer and shows that splice-switching ASOs targeting AURKA 5'UTR splicing disrupts this circuit, reducing cell viability and inducing apoptosis.

The paper shows that combining an ASO with HDAC inhibitors enhances SMN2 exon 7 inclusion in SMA mice, improving SMN expression and function.

This study develops a cocktail of ASOs to specifically increase CFTR W1282X mRNA and protein levels by inhibiting NMD, improving chloride current in human bronchial epithelial cells.

This paper develops an exon-skipping antisense oligonucleotide (ASO) strategy to increase CFTR protein levels in patients with the W1282X mutation by bypassing nonsense-mediated mRNA decay.

The paper systematically characterizes short intronic splicing-regulatory elements by analyzing their effects on exon splicing using a minigene approach.

The paper develops a computational method, mCross, to model RBP binding specificity using precise crosslink site registration and validates it with ENCODE eCLIP data.

This study demonstrates that GalNAc-conjugated ASOs can target cancer cells expressing ectopic ASGP-R, improving splice-switching ASO potency in vitro and in tumor models.

The paper identifies a human-specific switch in AFMID isoforms linked to TP53 mutations and HCC recurrence through splicing changes analyzed in RNA-Seq datasets.

The paper identifies de novo intronic indels in autism target genes from simplex families and finds they contribute to at least 10% of simplex autism.