The National Ataxia Foundation (NAF) is pleased to announce that 24 promising ataxia research studies, totaling more than $1,000,000, from the United States, Italy, Australia, United Kingdom, Portugal, and Germany were awarded funding at the December 2013 NAF Board of Directors meeting for fiscal year 2014. A listing and summaries of these studies are now available on the Foundation’s web site, http://www.ataxia.org/research/naf-research-2014.aspx.
This year’s NAF Scientific Review Panel reviewed 81 ataxia research proposals from 13 countries. The National Ataxia Foundation offers five international ataxia research programs including: the NAF Research “Seed-Money” Award, the NAF Post Doc Fellowship Research Award, the NAF Young Investigator Award, the NAF $50,000 Young Investigator SCA Award, and the $100,000 Pioneer SCA Translational Research Award.
Below is a listing of the funded ataxia research studies for fiscal year 2014:
RESEARCH SEED MONEY AWARDS
Codazzi, Franca, Ph.D.
San Raffaele Scientific Institute, Milano, Italy
IPSC-derived neurons from Friedreich's ataxia (FA) patients as a model to characterize pathological mechanisms and devise neuroprotective strategies
Fairbrother, Will, Ph.D.
Brown University, Providence, RI
Annotation and Analysis of Pre-mRNA Splicing Elements in Ataxia Genes
Houlden, Henry, Ph.D.
University College London Institute of Neurology, London, UK
Unraveling expression profiles in human SCA3 brain pathology: A pilot study
Mantuano, Elide, Doctor of Biology
National Research Council of Italy, Institute of Translational Pharmacology, Rome, Italy
Episodic ataxias: looking for new genes by next generation sequencing approach
Liliana Simões Mendonça, Ph.D.
University of Coimbra, Portugal
Transplantation of neural stem cells as a new therapeutic strategy for Machado-Joseph disease
Nobre, Rui Jorge, Ph.D.
University of Coimbra, Portugal
Silencing Machado Joseph-Disease/Spinocerebellar ataxia type 3 through the systemic route
Nobrega, Clevio, Ph.D.
University of Coimbra, Portugal
The role of ataxin-2 in is repeated twice Machado-Joseph disease: a molecular therapy approach with viral vectors
Rance, Gary, Ph.D.
University of Melbourne, Australia
The Effect of Cerebellar Abnormality on Hearing in Individuals with Spinocerebellar Ataxia (SCA) and Friedreich Ataxia (FRDA)
Schmahmann, Jeremy, M.D.
Massachusetts General Hospital, Boston, MA
Brief Ataxia Rating Scale for children (BARS-c)
Perlman, Susan, M.D.
University of California, Los Angeles
National Ataxia Database
YOUNG INVESTIGATOR AWARDS
Christie, Jason, Ph.D.
Max Planck Florida Institute, Jupiter, FL
Cellular signaling mechanisms underlying EA1 in cerebellum
Urban, Alexander, Ph.D.
Stanford School of Medicine, Palo Alto, CA
Genomic and epigenomic analyses in an induced pluripotent stem cell model of ADCA-DN
POST-DOC FELLOWSHIP AWARDS
Avery, Adam W. Ph.D.
University of Minnesota, Minneapolis
Dendritic transport dysfunction in a novel Drosophila model of SCA5
(Partnership with BAARC)
Li, Jian, Ph.D.
Northwestern University, Chicago, IL
Regulation of organismal proteostasis by integrated stress-response networks
Mollema, Nissa, Ph.D.
University of Minnesota, Minneapolis
CRISPR-modified ATXN1 in SCA1 Patient-derived iPS Cells
(Partnership with BAARC)
Yao, Bing, Ph.D.
Emory University, Atlanta, GA
5hmC-mediated Epigenetic Modulation in rCGG-mediated neurodegeneration associated with FXTA
$100,000 PIONEER SCA TRANSLATIONAL GRANT AWARDS
Davidson, Beverly, Ph.D.
University of Iowa, Iowa City, IA
Translating RNAi therapy for Spinocerebellar Ataxia 1 (SCA1) to the clinic
de Almeida, Luís Pereira, Ph.D.
University of Coimbra, Portugal
Pharmacological activation of autophagy to alleviate Machado-Joseph disease
LaSpada, Albert, M.D., Ph.D.
University of California, San Diego
Ataxin-7 knock-down therapy to treat SCA7: Preclinical trial validation in mice
Bezprozvanny, Ilya, Ph.D.
UT Southwestern Medical Center, Dallas, TX
Preclinical development of NS13001 for SCA2
$50,000 YOUNG INVESTIGATOR SCA RESEARCH AWARDS
Chen, Dong-Hui, M.D., Ph.D.
University of Washington, Seattle, WA
Pathogenesis and disease course of spinocerebellar ataxia type 14 in a mouse model
Koch, Philipp, M.D.
University of Bonn, Germany
Analysis of transcriptional changes in Machado-Joseph-Disease using isogenic patient-derived neurons
Lim, Janghoo, Ph.D.
Yale University, New Haven, CT
Molecular pathogenesis studies of spinocerebellar ataxia type 1
Zhang, Yong, Ph.D.
UMass Medical School, Worcester, MA
The role of Ataxin-2 in the regulation of circadian behavior and sleep in mice
The funding for these important ataxia research projects have been made possible through the generosity of NAF donors and partners who contributed to the 2013 NAF Annual Ataxia Research Drive, the $150,000 matching research grant from The Gordon and Marilyn Macklin Foundation, our corporate and foundation friends, the Michael and Patricia Clementz Family Endowment Fund for SCA 3 Research, NAF chapters and support groups, individual and group donations, individuals and families who conducted fund raising events, NAF Walk n’ Rolls for Ataxia events, the Bob Allison Ataxia Research Center (BAARC) and to our anonymous donor who made a three year research commitment totaling $1.5 million to support spinocerebellar ataxia research.