Pediatric cancer: Can CRISPR gene editing help?

[Simone T. Sredni, associate professor of pediatric neurosurgery at Northwestern University Feinberg School of Medicine] said that after so many years in the field, she has “never felt so close” to finding a better treatment for atypical teratoid/rhabdoid tumors (AT/RT) —and the powerful gene-editing tool CRISPR/Cas9 helped her get there.

AT/RT are very aggressive and unresponsive tumors that most frequently develop in very young children, under three months old.

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So, the idea came about to screen the kinome, which is the complete set of protein kinases encoded in the genome.

“Kinases are good therapeutic targets because they are involved in many cellular functions including proliferation and survival, and they can be targeted using inhibitors,” Sredni said.

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So her team mutated each one of the 160 kinases in a cell line derived from one of the very aggressive pediatric tumors and observed that mutations in 5 percent of them significantly impaired cell proliferation.

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The team has now moved on to studies in mice with intracranial xenograft.  While this is still under development, Sredni said it looks like tumors are actually growing significantly slower or even shrinking.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Using CRISPR to Find Treatments for Aggressive Pediatric Brain Cancer

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