We conducted an in vivo genome‐wide CRISPR‐Cas9 knockout screen using MTMCT‐derived cells to identify genes essential for tumor growth. SOD1 emerged as a key therapeutic target, and its inhibition suppressed tumor proliferation by disrupting redox homeostasis, both in vitro and in PDX models. ABSTRACT Malignant transformation of mature cystic teratoma (
Satoshi Tamauchi +8 more
wiley +1 more source
Non‐coding RNAs (ncRNAs) regulate breast cancer radioresistance via cell cycle, DNA repair and tumour microenvironment pathways. Targeting ncRNAs (e.g., HOTAIR and miR‐155) with RNA‐based therapies (ASOs and CRISPR) shows promise but faces delivery challenges.
Xiaohui Zhao +8 more
wiley +1 more source
Antisense oligonucleotide binding to U5 snRNP induces a conformational change that exposes the conserved loop of U5 snRNA [PDF]
Gil Ast
openalex +1 more source
LCN2 mediates the suppression of C2C12 myoblast proliferation and myotube formation via erastin‐induced ferroptosis. Mechanistically, the LCN2‐ACOD1 axis regulates skeletal muscle growth via mitochondria‐associated ferroptosis, providing a theoretical basis for the prevention and therapy of muscle‐related diseases.
Xiaojing Hao +12 more
wiley +1 more source
RFFL antisense oligonucleotides: A novel stabilization strategy to broaden the reach of CFTR modulator therapy. [PDF]
Pedemonte N.
europepmc +1 more source
Phosphatase PPP1CC Regulates the First Lineage Segregation by GAS5 in Mouse Preimplantation Embryos
Subcortical GAS5 directs PPP1CC phosphatase spatiotemporal positioning during mouse morula‐blastocyst transition, controlling YAP dephosphorylation to drive first embryonic lineage specification. ABSTRACT The transcriptional effector of the Hippo signalling pathway, YAP, regulates the first lineage specification in mouse preimplantation embryos ...
Jianwu Wang +10 more
wiley +1 more source
Artificial Intelligence-Driven Design of Antisense Oligonucleotides for Precision Medicine in Neuromuscular Disorders. [PDF]
Leckie J, Wu S, Standell T, Yokota T.
europepmc +1 more source
Mind the Gapmer: Implications of Co-transcriptional Cleavage by Antisense Oligonucleotides [PDF]
David G. Maranon, Jeffrey Wilusz
openalex +1 more source
Regulation of PHOX2B gene expression by the long non‐coding natural antisense RNA PHOX2B‐AS1
PHOX2B is a transcription factor essential for autonomic nervous system development. We identify and characterize PHOX2B‐AS1, a human long non‐coding antisense transcript at the PHOX2B locus, along with its murine counterpart. Our findings reveal bidirectional transcription and reciprocal regulation: PHOX2B activates PHOX2B‐AS1, whereas PHOX2B‐AS1 ...
Simona Di Lascio +12 more
wiley +1 more source
Antisense oligonucleotides to KIF1A polymorphisms expand targets and rescue patient-derived neurons in vitro. [PDF]
Zuccaro MV +13 more
europepmc +1 more source

