Single Antisense Oligonucleotides Correct Diverse Splicing Mutations in Hotspot Exons

Medicine, Basic Medical Sciences, Oncology, Life Sciences, Cell Biology, Biochemistry, Bioengineering, Genetics,
antisense oligonucleotide   splicing mutation   hotspot exon   RNA splicing   actionable genes   gene mutation   therapeutic strategy  

Broad-Spectrum Correction of Splicing Mutations in Rare Disease Hotspot Exons by Single Antisense Oligonucleotide: Review of a Recent 2025 PNAS Study I. Academic Background: The Challenge of Disease-Associated Splicing Mutations and Dilemmas in Antisense Therapy RNA splicing is a crucial step in the regulation of gene expression in eukaryotes. The ...

ILC2 Instructs Neural Stem and Progenitor Cells to Potentiate Neurorepair After Stroke

Medicine, Neurology, Life Sciences, Immunology,
neurorepair   ILC2   neural stem cells   stroke   neural progenitor cells   brain injury   therapeutic strategy  

Academic Background Stroke is one of the leading causes of adult disability globally, with the core issue being neuronal damage and neurological dysfunction. Although neurogenesis and neurorepair after stroke are considered crucial for recovery, the specific mechanisms remain incompletely understood. Recent studies have shown that immune cells play...

EVA1-Antibody Drug Conjugate as a Novel Therapeutic Strategy for Eliminating Glioblastoma-Initiating Cells

Medicine, Neurology, Oncology, Life Sciences, Immunology,
glioblastoma   EVA1   antibody drug conjugate   tumor-initiating cells   therapeutic strategy  

Background Introduction Glioblastoma (GBM) is one of the most aggressive brain cancers, with a median survival of approximately 15 months. Despite the use of multimodal treatments including surgery, chemotherapy, and radiotherapy, the overall survival rate of GBM patients has not significantly improved over the past few decades. Recent studies have...

The Alcatraz-Strategy: A Roadmap to Break the Connectivity Barrier in Malignant Brain Tumours

Medicine, Neurology, Neurosurgery, Oncology,
malignant brain tumours   cellular networks   supramarginal resection   tumour microtubes   cell-to-cell connections   therapeutic strategy   gliomas  

Cellular Network Connectivity and Therapeutic Strategies in Malignant Brain Tumors Academic Background Malignant brain tumors, particularly glioblastoma, are among the most aggressive and lethal tumors in the central nervous system. Despite significant advancements in tumor biology and treatment methods in recent years, the median survival for glio...

MYC-Dependent Upregulation of the De Novo Serine and Glycine Synthesis Pathway is a Targetable Metabolic Vulnerability in Group 3 Medulloblastoma

Medicine, Neurology, Neurosurgery, Oncology,
medulloblastoma   MYC amplification   metabolic vulnerability   serine synthesis   glycine synthesis   PHGDH inhibition   therapeutic strategy  

Targeting the Serine/Glycine Synthesis Pathway in MYC-Driven Medulloblastoma Background Medulloblastoma is one of the most common malignant brain tumors in children, accounting for a significant proportion of childhood cancer deaths. Based on molecular characteristics, medulloblastoma is classified into four major subgroups: WNT, SHH, Group 3 (MBGr...