CRISPR Uncovers New Gene Fusion as Novel Drug Target for Brain, Ovarian and other Cancers


CRISPR-Cas9, the genome editing tool, has caused quite a stir in the scientific community. According to a recent article in Science Daily, with results published in Nature Communications, CRISPR has enormous potential. It is cheaper, faster, more versatile, and also more accurate than tools that have been used previously to edit DNA.

Medical researchers and geneticists are now able to remove, add or alter sections of the DNA sequence.

Why Edit DNA?

One aspect of gene editing occurs when a microbial cell is infected by a virus. The cell identifies the damage and attempts to repair it. The microbe uses the enzyme Cas9 to chop off and store parts of the viral DNA. Scientists are able to use such DNA repair machinery to make changes to one or more genes in a target cell.

The DNA fragment is stored with a genetic memory that allows the disabling of the same strain of infections in the future.

It is believed that CRISPR will bring about many changes in our daily lives.

About Gene Fusions

Gene fusions are used to determine a cancer patient’s response to a drug. They are also used as a diagnostic tool to predict a patient’s chances of overall survival.

A number of cancer treatments are now targeting gene fusions, which are the result of two previously separated genes. Scientists agree that gene fusions are perfect for diagnostic purposes as they can identify a subclassification of what had seemed to be identical disease entities.

Many gene fusions provide information about risk factors. Chimeric proteins that are encoded by the gene fusions can be targeted for treatment.

Impressive Numbers

To date, researchers have located approximately 20,000 gene fusions but were unable to determine their function in cancer development.

OpenTargets is the first major study of the function of gene fusions. The study analyzes which gene fusions may be responsible for the growth of cancer cells.

OpenTargets analyzed over 8,000 gene fusions that were taken from over 1,000 human cancer cell lines in 43 cancer types.

The research team used these cell lines to test against over 350 anti-cancer drugs in an attempt to determine which drugs could be repurposed. Their goal was to use these repurposed drugs to treat cancer patients who carried specific gene fusions.

CRISPR was employed as a means of discovering the primary gene fusions that are key to cancer cell survival.

The results showed that 90% of gene fusions are not at all cancer-related. Therefore, most gene fusions do not play a role in cancer cell survival.

About YAP1-MAML2 and other Gene Fusions

The researchers discovered YAP1-MAML2, a gene fusion that will become a drug target for ovarian and several other cancers.

The study also produced results that point to gene fusions relating to RAF1, ROS1, and BRD4 as targets for drugs already developed. This presents treatment options for patients who have sub-types such as breast, lung and pancreatic cancers.

The findings of the OpenTargets study will be part of the Cancer Dependency Map which is a rulebook for future cancer treatment. Results of the study confirm the use of certain gene fusions for guidance in the treatment of cancer patients.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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