Peter Mac scientists develop CRISPR method to target gene fusions in one in five cancers

Peter Mac researchers have developed a programmable CRISPR-Cas13b method to target and silence gene fusions, which drive approximately 20% of all human cancers. In laboratory cell models led by Dr. Wenxin Hu and Dr. Mohamad Fareh, the system successfully silenced nine distinct fusion types, including BCR-ABL1 in leukemia and EWSR1-FLI1 in Ewing's sarcoma. While offering personalized cancer therapy, the approach remains untested in animals and humans, pending effective tumor delivery methods.

Development and study publication

• ▪Peter Mac researchers developed a CRISPR-Cas13b nuclease method to precisely target and silence abnormal gene fusion transcripts in cancer cells.
• ▪Peter Mac researchers Dr. Wenxin Hu and Dr. Mohamad Fareh successfully targeted and silenced nine distinct gene fusions, including BCR-ABL1, in laboratory cell models.
• ▪The proof-of-concept study on PspCas13b gene fusion silencing was published in Science Advances in October 2026 by lead author Dr. Wenxin Hu and colleagues.

Personalized targeting mechanism

• ▪Peter Mac researcher Dr. Mohamad Fareh stated that the long-term vision is developing programmable cancer medicines tailored to the specific genetic abnormalities driving an individual tumor.
• ▪The CRISPR-Cas13b treatment strategy offers personalized cancer therapy by tailoring guide sequences to match an individual patient's specific tumor fusion pattern.
• ▪The CRISPR-Cas13b system is reprogrammed to recognize unique molecular breakpoint sequences formed when two distinct genes fuse together inside a cell.

Hurdles to clinical translation

• ▪The CRISPR-Cas13b gene fusion silencing technique has been demonstrated only in laboratory cell cultures and remains untested in animal models or humans.
• ▪Determining how effectively CRISPR machinery can be delivered into living tumors represents a primary untested hurdle for the research team.
• ▪Researchers must verify that CRISPR-Cas13b can selectively target cancer cells without damaging healthy cells in complex living disease models.

Role of gene fusions in cancer

• ▪Gene fusions occur when cellular DNA breaks and undergoes faulty repair, abnormally joining parts of two distinct genes together.
• ▪According to research published in Science Advances, gene fusions are present in approximately 20% of all human cancers and act as direct oncogenic drivers in 16.5% of cancers.
• ▪The BCR-ABL1 gene fusion is present in nearly all chronic myeloid leukemia cases, while the EWSR1-FLI1 fusion occurs in over 85% of Ewing's sarcoma cases.