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Published on: June 23, 2023
Genetically engineered mouse models of brain cancer and the promise of preclinical testing
1Department of Cancer Biology and Genetics, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
Abstract:
Recent improvements in the understanding of brain tumor biology have opened the door to a number of rational therapeutic strategies targeting distinct oncogenic pathways. The successful translation of such "designer drugs" to clinical application depends heavily on effective and expeditious screening methods in relevant disease models. By recapitulating both the underlying genetics and the characteristic tumor-stroma microenvironment of brain cancer, genetically engineered mouse models (GEMMs) may offer distinct advantages over cell culture and xenograft systems in the preclinical testing of promising therapies. This review focuses on recently developed GEMMs for both glioma and medulloblastoma, and discusses their potential use in preclinical trials. Examples showcasing the use of GEMMs in the testing of molecularly targeted therapeutics are given, and relevant topics, such as stem cell biology, in vivo imaging technology and radiotherapy, are also addressed.
Insights
Genetically engineered mouse models (GEMMs) offer advanced preclinical testing for brain cancer therapies by mimicking tumor genetics and microenvironments. These models are crucial for developing targeted drugs for glioma and medulloblastoma.
Area of Science:
- Neuro-oncology
- Cancer Biology
- Preclinical Research
Background:
- Advances in brain tumor biology enable targeted therapies.
- Effective preclinical models are essential for translating these therapies into clinical use.
- Genetically engineered mouse models (GEMMs) closely mimic human brain tumors, including their genetic makeup and microenvironment.
Purpose of the Study:
- To review recently developed GEMMs for glioma and medulloblastoma.
- To discuss the utility of GEMMs in preclinical drug testing.
- To highlight the integration of GEMMs with other research areas like stem cell biology, in vivo imaging, and radiotherapy.
Main Methods:
- Focus on genetically engineered mouse models (GEMMs) for brain tumors.
- Review of studies utilizing GEMMs for preclinical therapeutic screening.
- Discussion of comparative advantages of GEMMs over cell cultures and xenografts.
Main Results:
- GEMMs provide a more accurate recapitulation of brain tumor complexity compared to traditional models.
- GEMMs facilitate the testing of molecularly targeted therapeutics.
- Recent developments in GEMMs offer improved platforms for studying brain cancer.
Conclusions:
- GEMMs are valuable tools for the preclinical evaluation of novel brain cancer treatments.
- The use of GEMMs can accelerate the development of effective therapies for glioma and medulloblastoma.
- Integrating GEMMs with advanced technologies enhances their utility in neuro-oncology research.
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