Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University

Cure SMA has awarded a $150,000 research grant to Laxman Gangwani, PhD, at the Texas Tech University Health Sciences Center, for his project entitled, “Function of Senataxin as a Protective Modifier of Spinal Muscular Atrophy”.

Dr. Gangwani and his team will study how the expression of the Senataxin protein may serve as a protective genetic modifier of SMA disease severity. They hypothesize that increased amounts of Senataxin will decrease SMA symptoms in a mouse model of disease. If validated, the study results will help establish Senataxin as a potential novel therapeutic target and will promote further mechanistic studies into how Senataxin is able to modify SMA symptoms.

Meet Dr. Gangwani

Who are you?

I am an associate professor in the Department of Biomedical Sciences at the Paul L. Foster School of Medicine at the Texas Tech University Health Sciences in El Paso, Texas. A cell and molecular biologist by education and training, I received my Ph.D. from the University of Delhi, India and my postdoctoral training at the University of Massachusetts School of Medicine. My areas of expertise include creation of novel mouse models to delineate the function of protein-protein complexes and the molecular mechanisms of neurodegeneration associated with neuromuscular disorders, including SMA.

How did you first become involved with SMA research?

About 20 years ago, I discovered that the zinc finger protein, ZPR1, interacts with the survival motor neuron (SMN) protein and is required for accumulation of SMN in the nucleus of cells. The interaction of SMN with ZPR1 was disrupted in SMA patients. These initial findings were published in 2001 and set the stage for me to continue research in the field of SMA. I am proud to state that one of my first research grants on SMA was funded by the Families of SMA (now Cure SMA).

What is your current role in SMA research?

My lab focuses on the cellular and molecular mechanisms of neurodegeneration associated with the pathogenesis of SMA. We identify and characterize new molecular targets that contribute to motor neuron degeneration. We validate molecular targets as potential therapeutic targets by using genetic and pharmacological methods, including creation of new SMA mouse models. We establish the role of potential drug targets as SMN-dependent or SMN-independent modifiers to help develop suitable stand-alone and combination therapies for the treatment of a broad spectrum of SMA patients with varying disease severity.

What do you hope to learn from this research project?

We hope to learn if and Senataxin protein functions as a protective modifier of SMA and develop a novel (SMN-independent) therapeutic strategy for the treatment of SMA.

How will this project work?

We will create and characterize a novel genetically modified mouse which overexpressed the Senataxin protein. We will cross this new mouse model with an SMA mouse model and look for improvement in SMA disease in these mice.

What is the significance of your study?

Findings from this study will provide a genetic proof-of-concept and will establish Senataxin protein as a viable SMN-independent therapeutic target that could be used as a stand-alone treatment or in combination with SMN-dependent treatments for SMA.

Basic Research Funding

This grant to Dr. Gangwani 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.

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