Research Funding

National Institutes of Health

NeuroTherapeutics Research Consortium (NTRI)
The principal objective of the Research Consortium is implementation of a highly integrated, interdisciplinary approach to develop targeted molecular therapeutics for neurogenetic disorders, using the late-onset neurodegenerative disorder fragile X-associated tremor/ataxia syndrome (FXTAS) as its principal research paradigm. The fundamental challenge addressed by the Consortium is how to develop and integrate the various components required to achieve (and measure) a targeted therapeutic response to a CNS disorder.

Development of Targeted Therapeutic Agents for the Treatment of FXTAS (NTRI)
The principal objective of this project will be the identification and assessment of various candidate therapeutic agents that might attenuate the effects of the pathogenic RNA in FXTAS. Research will also involve further study of neural cell models of FXTAS.

Fragile X-associated Tremor/Ataxia Syndrome
The principal objective of the proposed research is the identification of the mechanistic basis for a progressive neurological disorder, fragile X-associated tremor/ataxia syndrome (FXTAS), which involves intention tremor, gait ataxia, and dementia, and affects at least one-third of males over fifty years of age who carry small (premutation, CGG-repeat) expansions of the fragile X mental retardation 1 (FMR1) gene.

Expression of the Fragile X Gene
This project is designed to examine the mechanisms underlying both the silencing of the FMR1 gene and the increase in FMR1 mRNA observed for carriers of premutation FMR1 alleles.

Genotype-Phenotype Relationships in Fragile X Families
The overall focus of this project is to explore both the neurodevelopmental and neurodegenerative effects of the fragile premutation in both children and adults.

Social-Affective Bases of Word Learning in Fragile X Syndrome and Autism
The overall focus of this UC Davis and University of Wisconsin Waisman Institute collaboration is the study of the behavioral and cognitive phenotype of fragile X syndrome (FXS) with autism and to investigate the neurological and molecular basis of this phenotype.

Foundation Funding

Private gift funds

The Hagerman laboratory has been supported in part by generous gifts/donations by individuals or families that have a specific interest in fragile X or FXTAS research, or who see the potential of our research to shed additional light on other neurodevelopmental (e.g., autism) or neurodegenerative (e.g., Alzheimer or Parkinson) disorders. Such funds, which are generally unrestricted, are the most effective means for rapidly initiating promising new research directions. For example, our original discovery of elevated FMR1 mRNA levels in premutation carriers was made solely through donations from families.