Related Experiment Video

Updated: Sep 27, 2025

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

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Targeting mutations in cancer

Michael R Waarts1,2,3,4, Aaron J Stonestrom2,3,4,5, Young C Park2,3,4

  • 1Gerstner Sloan Kettering Graduate Program in Biomedical Sciences.

The Journal of Clinical Investigation
|April 15, 2022
PubMed

Insights

Targeted cancer therapies, driven by genetic sequencing, offer improved outcomes for some cancers. Further research is needed to maximize benefits for all patients with targetable mutations.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Targeted therapies have become crucial in cancer treatment over the last 20 years.
  • Advances in sequencing technology have enhanced understanding of cancer's mutational landscape and genetic drivers.
  • Tumor genetic testing is now vital for selecting appropriate targeted therapies.

Purpose of the Study:

  • To review current knowledge on targetable mutations across various cancers.
  • To highlight key examples of druggable mutations and their targeting strategies.
  • To discuss challenges in maximizing the therapeutic benefits of targeted cancer therapies.

Main Methods:

  • Literature review of targeted therapies in oncology.
  • Analysis of genetic sequencing data and mutational landscapes.
  • Examination of clinical trial outcomes and research challenges.

Main Results:

  • Targeted therapies have significantly improved outcomes in certain cancers.
  • Efficacy of targeted approaches varies across different oncologic contexts.
  • Numerous targetable mutations are being identified in hematologic malignancies and solid tumors like lung and breast cancer.

Conclusions:

  • Targeted therapies represent a significant advancement in cancer treatment.
  • Continued research is essential to overcome challenges and expand the efficacy of targeted therapies.
  • Understanding tumor-specific mutations is key to personalized cancer treatment strategies.

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