Related Experiment Video
Updated: Oct 9, 2025

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Targeting RAS in neuroblastoma: Is it possible?
Lei Lin1, Lei Miao1, Huiran Lin2
1Department of Pediatric Surgery, Guangzhou Institute of Pediatrics, Guangdong Provincial Key Laboratory of Research in Structural Birth Defect Disease, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou 510623, Guangdong, China.
RAS mutations are common in recurrent neuroblastoma, driving tumor growth via the MAPK pathway. Targeting these RAS mutations offers a promising personalized therapy approach for difficult-to-treat pediatric cancers.
Area of Science:
- Pediatric Oncology
- Molecular Biology
- Cancer Genetics
Background:
- Neuroblastoma is a common childhood cancer with complex pathogenesis.
- Genetic heterogeneity limits current monotherapy efficacy.
- RAS mutations are increasingly recognized in recurrent neuroblastoma.
Purpose of the Study:
- To review the biological role of RAS in neuroblastoma.
- To summarize the frequency of RAS mutations in neuroblastoma.
- To discuss therapeutic strategies targeting RAS mutations in neuroblastoma.
Main Methods:
- Literature review of studies on RAS mutations in neuroblastoma.
- Analysis of preclinical and clinical data for RAS-targeted therapies.
- Exploration of the MAPK pathway's role in RAS-driven neuroblastoma.
Main Results:
- RAS mutations activate the MAPK pathway, crucial for neuroblastoma development.
- Targeting RAS mutations shows promise in preclinical models.
- Existing therapies for RAS-mutated tumors offer insights for neuroblastoma treatment.
Conclusions:
- RAS mutations represent a significant therapeutic target in neuroblastoma.
- Personalized therapies targeting RAS are essential for improving outcomes.
- Further research and clinical trials are needed to overcome challenges in treating RAS-mutated neuroblastoma.
Related Concept Videos
The Ras Gene
Ras is a...
Small GTPases - Ras and Rho
Three regulatory proteins control their activity:
The Retinoblastoma Gene
The first-ever tumor suppressor gene called Rb was identified in retinoblastoma - a rare eye tumor in children. In inherited forms of the disease, a child inherits one defective copy of the Rb gene, which predisposes them to retinoblastoma. However,...
Targeted Cancer Therapies
There are several types of targeted therapies against...
Abnormal Proliferation

