Table 2 Related therapeutic targets: targeting specific RBP

From: Alternative splicing and related RNA binding proteins in human health and disease

Drugs

Target

Therapeutics

Reference

VPC-80051

HNRNPA1

VPC-80051 directly interacts with HNRNPA1 RBD and reduces the level of AR-V7 mRNA in the 22Rv1 CRPC cell line

492

SPHINX31

SRPK1

Inhibits the phosphorylation of SRSF1 and promotes angiogenesis caused by VEGF-A isoforms.

493

Tasisulam, Chloroquinoxaline, sulfonamide, Indisulam

RBM39

Promotes RBM39 recruitment to the CUL4-DCAF15 E3 ubiquitin ligase, leading to multi-ubiquitination and proteasomal degradation of RBM39

494

SM09419

CLK/DYRK

Causes erroneous splicing and inactivation of the apoptosis inhibitor XIAP, downregulating the anti-apoptotic protein BCL2A1 related to venetoclax resistance

295

Auranofin

NONO

Regulates the abnormal ASE of GPX1, inhibiting tumor growth, invasion, and redox balance

221

TG003

SRSF1

Inhibits the activity of Clk1/4, leading to the dephosphorylation of SFRS1, thereby inducing the subcellular relocalization of SFRS1 and inhibiting the ASE of SFRS1-dependent pre-mRNA.

495

TG693

SRSFs

Inhibits the phosphorylation of SRSFs that are substrates of CLK1, and regulates pre-ribonucleic acid splicing in skeletal muscle

496

T025

SRSFs

Inhibits the phosphorylation of SRSFs that are substrates of CLK2, inducing exon skipping of pre-mRNA AS regulated by SRSF1

497

PRMT5-IN-31

PRMT5/HNRNPE1

Acts as PRMT5 inhibitor/HNRNPE1 upregulator

498

Manumycin-A

Ras signaling, HNRNPH1

Inhibits the Ras signaling pathway and HNRNPH1 expression to suppress the biogenesis and secretion of CRPC cell exosomes

499

Riluzole

HNRNPA1

Inhibits IRES-dependent translation, and blocks the binding of HNRNPA1 with cyclin D1 and MYC IRES, thereby significantly reducing the translation efficiency of these transcripts

500

JL014

HNRNPE1

Increases the mRNA and protein levels of HNRNP1 in HUVEC

501

Spinraza

HNRNPA1

Blocks the intron binding site of HNRNPA1, promotes the inclusion of exon 7 of SMN2 and the level of full-length SMN2 mRNA, treating SMA

502