MicroRNA Breakthroughs: Paving the Way for Vision Restoration
Research News
Researchers funded by the Foundation Fighting Blindness are developing microRNA-based therapies for inherited retinal diseases, building on recent Nobel Prize-winning discoveries.
The Foundation Fighting Blindness is thrilled to share that our funded researchers are leveraging groundbreaking discoveries in microRNA, recently honored with the 2024 Nobel Prize in Physiology or Medicine.
Nobel Laureates Victor Ambros and Gary Ruvkun have unveiled the crucial role of microRNA in gene regulation. Their pioneering work revealed that microRNAs, small non-coding RNA molecules, play a vital role in controlling gene expression by binding to messenger RNAs and preventing them from producing proteins. This discovery has opened new avenues for understanding and treating various diseases, including inherited retinal diseases (IRDs).
Building on this, our esteemed researchers, Dr. Alberto Auricchio, Dr. Sandro Banfi, and Dr. Deniz Dalkara, are pioneering microRNA-based therapies for retinal diseases through their program, “RetMir.” Dr. Auricchio and co-investigators are identifying microRNAs and microRNA antagonists or ‘sponges’ that have a neuroprotective effect on the retina in order to provide a one-fits-all, mutation-independent therapeutic approach for inherited retinal degenerations. The team is screening hundreds of microRNAs to identify one or more microRNAs that either alone or in combination protect against retinal degeneration in different models of inherited retinal degeneration. If this project is successful, it will provide a gene therapy for a variety of IRDs that are currently untreatable and for which conventional augmentation methods may not be available.
Additionally, Dr. Simon Petersen-Jones at Michigan State University is exploring gene knock-down strategies using artificial microRNA to treat dominant CRX-associated retinal diseases (autosomal dominant LCA). A single bad copy of CRX can cause diseases. This strategy will stop the bad copy of CRX and add back a good copy of CRX using artificial microRNA that will also be microRNA resistant. Dr. Petersen-Jones is looking to test this knockdown and replace technique on CRX as a proof of concept that can also be modified for other dominantly inherited diseases.
Together, these efforts represent significant development towards treatments and cures for blinding diseases!
