Overview
Leveraging SMN role in translation to develop the next-gen of biomarkers for SMA
- Principal investigator(s):
- Dr. Gabriella Viero
- Institution:
- National Research Council of Italy (CNR)
- Grant:
- €121,500
- Grant Type:
- Operating Grant
- Start Year:
- 2024
- Duration:
- 2 Years
- Call number:
- 12
- Status:
- Ongoing
Dr. Gabriella Viero
Dr. Viero is the Principal investigator of a laboratory she established in 2011 at the Institute of Biophysics, CNR Italy. Her research focuses on studying translational control of gene expression and molecular mechanisms underlying Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis. The lab is seeking to move forward strong interdisciplinary approaches, combining expertise in biophysics, biochemistry, mathematics, computational biology, neuropathology and cell biology. She has obtained several grants from national, European and American funding agencies and foundations. Her research has led to the publication of several papers in high-impact journals.
In Focus
Background
While the development of treatments increasing SMN levels marks significant progress in managing SMA, challenges persist and new symptoms are emerging in treated individuals that affect various bodily functions beyond muscles and nerves. This underscores the need for a broader understanding of SMA as a systemic condition. As existing biomarkers for SMA are limited and focus only on neuromuscular indicators, Dr. Viero and her team aim to develop advanced biomarkers, to track disease progression and treatment responses accurately.
How will Dr. Viero's team do this?
Dr. Viero’s previous work has showed how SMN loss affects protein translation in SMA models. Using advanced technologies, the team will now test samples from SMA patients treated with approved drugs. Their goals include confirming the role of SMN in protein production, identifying specific translational changes indicative of SMA subtypes, and validating these biomarkers in real-world patient samples.
Why is it interesting to patients?
This project aims to provide individuals living with SMA, their caregivers, and clinicians with a new class of biomolecular indicators for accurately assessing disease progression and predicting individual responses to therapy, ultimately improving outcomes for SMA patients.