New CRISPR Method Makes Protein Production in Cells Controllable
New Perspectives on Rare Diseases and Cancer
Advertisement
TAPIR specifically increases protein production—drawing on new insights into stem cell biology and disease-related processes.
How quickly a cell produces proteins plays a decisive role in determining whether it divides, differentiates, or retains its stem cell properties. A research team led by Professor Stefan H. Stricker, Professor of Epigenetic Engineering at the LMU Biomedical Center and group leader at Helmholtz Munich, in collaboration with international partners, has demonstrated for the first time that the amount of ribosomal RNA (rRNA) directly regulates these processes. Their findings have been published in the journal *Science*.
New Method Enables Targeted Control of Ribosomal RNA
It was previously known that the amount of ribosomal RNA varies among different cell types and is altered in numerous diseases. However, it remained unclear whether these differences were the cause or merely the consequence of biological processes.
With the newly developed CRISPR-based method TAPIR (Targeted Activation of Protein Translation), researchers now have a tool to increase the activity of ribosomal genes and thereby influence a cell’s protein production. “Our new study shows that targeted activation of rRNA production significantly increases protein synthesis,” explains Stricker, the study’s last author.
New Perspectives for Rare Diseases and Cancer
The findings could be particularly relevant for diseases in which ribosomal function is impaired. These include ribosomopathies such as Treacher-Collins syndrome, a rare congenital disorder that causes facial malformations. In a mouse model, the researchers succeeded in partially compensating for disease-related changes by specifically boosting rRNA production.
Furthermore, the research team observed that similar mechanisms also play a role in pancreatic cancer. Tumor cells apparently use increased rRNA production to sustain their rapid growth. In a mouse model of pancreatic cancer, TAPIR was able to increase rRNA production and promote the growth of cancer cells. This demonstrates that increased rRNA production causally contributes to tumor growth and is not merely a side effect.
A Platform for Further Research on Health-Related Topics
“Our study makes it clear that the regulation of protein biosynthesis plays a central role in both developmental and growth processes as well as in the development of cancer,” Stricker summarizes. He sees TAPIR as a research platform for better understanding the role of protein biosynthesis in health and disease and, in the long term, for developing new therapeutic approaches.
It is conceivable that this approach could be applicable in the future to diseases associated with impaired ribosomal function, as well as open up new therapeutic targets for treating tumors in which protein production has spiraled out of control.
Note: This article has been translated using a computer system without human intervention. LUMITOS offers these automatic translations to present a wider range of current news. Since this article has been translated with automatic translation, it is possible that it contains errors in vocabulary, syntax or grammar. The original article in German can be found here.
Original publication
Other news from the department science
