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Publications of the Week

Engineering, Delivery, and Biological Validation of Artificial microRNA Clusters for Gene Therapy Applications

By December 9, 2019December 16th, 2019No Comments

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This week we profile a recent publication in Nature Protocols from Dr. Pierpaolo Peruzzi (pictured, third from left) with his lab at Brigham and Women’s Hospital.

Can you provide a brief overview of your lab’s current research focus?

We are interested in exploring the yet untapped features of non-coding RNA biology and harness them to develop a platform for a new gene therapy approach. Our goal is to build RNA-based “drugs” to help cure brain cancer, one of the most formidable, devastating, and poorly responsive tumor.

What is the significance of the findings in this publication?

MicroRNAs are tiny RNAs belonging to the larger family of non coding RNAs (i.e. genes that are transcribed but do not become proteins) and have a crucial role in keeping cells healthy and in check.  Unsurprisingly, in cancer, many of them are dysregulated simultaneously, and each one partially contributes to the malignancy of the disease.  Our last publication  provides the research community with a “recipe” to build up artificial genes encoding multiple microRNAs of choice. This way, the biological effect of microRNAs can be multiplied  and harnessed to its fullest. Following this step-by-step protocol, researchers just have to cherry-pick their favorite microRNA combinations and in a few easy steps they can engineer their custom-made genes for their investigational and therapeutic interests, whatever they are.

 What are the next steps for this research?

We are currently deepening our understanding of RNA structure and processing, which allows us to increase the complexity and functionality of our transgenes. In parallel, we are working on the best solutions to deliver these artificial genes to patients, with the goal of  targeting the most crucial vulnerabilities of cancer. More to come soon!

If you’d like us to mention your funding sources, please list them.

  1. NIH 5K12NS80223
  2. NIH 1K08NS101091
  3. Department of Neurosurgery, Brigham and Women’s Hospital


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