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Families of Spinal Muscular Atrophy Awards New Research Funding of $140,000 to Dr. Custer in the Androphy Lab at Indiana University. 

December 21, 2012.

Description of FSMA Funded Project: The role of vehicle coat protein alpha-COP in new models of SMA.
This award supports Dr. Sara Custer's postdoctoral training in the lab of Dr. Elliot Androphy at the Indiana University School of Medicine. The main focus of the project is to understand how low levels of the SMN protein leads to dysfunction of motor neurons. To achieve this goal, the group will generate new cell culture and animal models of SMA to gain a better understanding of the basic functions of SMN protein and with novel cellular binding partners in motor axons. 
Research Strategy
: The researchers have identified a protein called alpha-COP that interacts with SMN and will investigate whether this interaction is necessary for normal maturation and function of motor neurons.  First, they have generated a cell culture model of SMN depletion, which can be used as a biological assay of SMN functions, such as those important for growth and maintenance of neurons. The cells will be used to determine which aspect of SMN and its various interacting partners are important for neurite outgrowth and normal cellular function. Second, they have used a novel viral-mediated transgenic approach to produce two new mouse lines that will help elucidate the importance of alpha-COP function to SMA pathology.

Earlier this year, FSMA's Advisory Boards met to evaluate new research funding for 37 basic research grant applications and 7 drug discovery projects for SMA. The organization is planning to award $1.4 Million in new research funding over the next few months. This new round of research funding will be allocated into three areas: 1) Basic Research to understand the disease and provide ideas for drug making, 2) Drug Discovery to develop new SMA therapies, and 3) Clinical Research to help test new drugs effectively and to improve care for patients.
Click here to see more details on the process.

Our research progress provides us all with hope that one day we will live in a world without SMA. Your gift will make that hope a reality.  Click here or the image below to donate and support Families of SMA research and services.

Researcher Profile:

Who are yDr.  Custerou?
I have a PhD from the University of Washington in Neurobiology and Behavior and I study hereditary neurodegenerative diseases in cell culture and mouse models.
How did you first become involved with SMA research?

I became involved with SMA after moving to Indianapolis and joining the research lab of Dr. Elliot Androphy.  I had previously worked with some type II and III patients at an amazing therapeutic equestrian facility in Woodinville, WA.  Equestrian therapy is great for your core and for your spirit!  Click here for the Androphy webpage.  
What is your current role in SMA research?
I am interested in learning more about the basic biology of SMA and how other proteins can influence the health and maintenance of motor neurons.  We use a combination of cell models and animal models to address these questions.  The more we know about the cellular environment in SMA, the more targets we have to aim at for therapeutic intervention.

Click here for our funding facts page for details on FSMA research awards. 

This grant to Dr. Custer at Indiana University will help us answer the key basic research question of what function does SMN protein perform in motor neurons.

The basic research that Families of SMA has funded, through 145 research grants to 75 institutions around the world, has delivered major discoveries:
-We know the cause of SMA. Which means we can develop treatments that correct the underlying cause of the disease rather than just reduce symptoms.
-We identified a back-up gene for SMA. Which means we have a straightforward drug target already in the body: a built-in switch for new therapies to work on.
Using this knowledge, we now have 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.


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