Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences

Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that they would commit their careers to developing a treatment and cure for SMA.

Cure SMA honors Audrey’s legacy with the Audrey Lewis Young Investigator Award, periodically given to younger researchers working in the SMA field. The goal is to make a positive impact on the early phase of a talented researcher’s career, enabling them to focus on the SMA field. 

Cure SMA is pleased to announce two 2019 recipients of the Audrey Lewis Young Investigator Award, the first of whom is Veronica Pessino, PhD, at the Salk Institute for Biological Sciences. Dr. Pessino is a post-doctoral researcher, working in the laboratory of Dr. Samuel Pfaff. She will receive $150,000 for her project, “Identifying SMN and microRNA-218 impacts on the local transcriptome of neuromuscular junctions.”

Dr. Pessino will study how the overexpression of a molecule, miR-218, that the Pfaff lab has previously shown to be involved in support of neuromuscular junctions, may be able to lessen the negative effects of low levels of SMN.

If validated, the study results will help establish miR-218 as a potential novel therapeutic target and will promote further mechanistic studies into how miR-218 is able to modify SMA symptoms.

Meet Dr. Pessino

Who are you?

I earned my PhD at the University of California, San Francisco, in the Biophysics department. There I studied adrenergic cell-signaling pathways while developing tools for improved super-resolution microscopy. Prior to earning my PhD, I earned a bachelor’s degree in Physics and Biology at the University of California, Santa Barbara, while researching the biophysical properties of Alzheimer’s implicated molecules.

How did you first become involved with SMA research?

I, like most people, have been personally affected by friends and family experiencing devastating neurodegenerative disorders. During my PhD, I became fascinated by the molecular basis driving these maladies and aimed to transition to a lab where I could focus on these challenges for my postdoctoral studies.

What is your current role in SMA research?

In general, the Pfaff lab focuses on determining neuronal circuitry in the spinal cord, while investigating motor-neuron diseases such as SMA and ALS, as well as neuronal injury. In this group, I aim to bring forward my previous expertise in cell biology and microscopy to further our understanding of the molecular basis of SMA.

What do you hope to learn from this research project?

We know the cause of SMA is the lack of a protein called survival motor neuron (SMN), however it is unclear why lack of this protein specifically hurts motor neurons and their ability to communicate with muscles through neuromuscular junctions (NMJs). The objective of this work is to understand SMN’s involvement in communication maintenance, and the mechanisms behind NMJ breakdown, while testing a potential remedy for improved NMJ communication when SMN levels are low in SMA patients.

How will this project work?

In order to understand NMJ maintenance, we must first identify what molecules are most important and locally enriched at that part of the motor neuron by (1) sequencing all the local transcripts from NMJs in cultured cells, and dissected out of mice, and (2) comparing the sequencing results from normal cells/mice to those that model SMA and are lacking the SMN protein. Previously, we identified a molecule, miR-218, that we showed is involved in NMJ support, and may be able to buffer the negative effects of missing SMN, so we’ll insert miR-218 in abundance into the cells and mice and quantify its effect on their survival.

What is the significance of your study?

Once we understand what maintains a functional NMJ, and how SMN protein is involved in that maintenance, we will have new molecules to target and potentially prevent NMJ breakdown from happening, and therefore, SMA from progressing. Furthermore, this work will characterize the effects of miR-218 abundance, and if they are positive, miR-218 could be a promising therapy to keep SMA patients from progressing in disease.

Basic Research Funding

This grant to Dr. Pessino is part of $1,150,000 in new basic research funding that we’re currently announcing.

Basic research is the first step in our comprehensive research model. We fund basic research to investigate the biology and cause of SMA, in order to identify the most effective strategies for drug discovery. We also use this funding to develop tools that facilitate SMA research.

Past Announcements

$150,000 Grant to Laxman Gangwani, PhD

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