Treatment Targets Archives • Page 6 of 7 • FRAXA Research Foundation

Glutamate Metabolism in Fragile X Mouse Brain

February 15, 2008

Dr. Mary McKenna’s FRAXA-funded study at the University of Maryland examined how mGluR activity impacts key brain pathways linked to Fragile X.

James Malter, at University of Wisconsin-Madison, FRAXA research grant

Using Fenobam to Reduce APP and Abeta in Fragile X Mice

February 14, 2008

With FRAXA funding, Drs. James Malter and Cara Westmark studied how APP and its metabolite may drive excess protein synthesis in Fragile X, aiming to restore balance.

AMPAkines and BDNF in Fragile X: UCI Researchers Restore Memory Process in Fragile X

February 14, 2008

FRAXA’s $104K grant supported Dr. Julie Lauterborn at UC in studying dendritic spines and memory-related treatment targets in Fragile X mice.

Targeting the Role of Group 1 Metabotropic Glutamate Receptors

February 14, 2008

Dr. Huibert Mansvelder’s FRAXA-funded study at the University of Amsterdam examined receptor responses to drugs, revealing new therapeutic insights.

Effects of Alternative Splicing at FMR1 Exon 15 on Understanding Fragile X Syndrome

February 14, 2008

FRAXA-funded research by Dr. Robert Denman explored how exon 15 affects FMRP protein splicing—offering insights into Fragile X molecular mechanisms.

In Vitro Reactivation of the Fragile X Gene

January 14, 2008

With FRAXA funding, Dr. Giovanni Neri’s team in Italy explored reactivating the FMR1 gene and studying cell lines with unmethylated full mutations.

Randall Carpenter, MD, at Massachusetts Institute of Technology, FRAXA research grant

FRAXA Contributes $10,000 to NIH grant to Seaside Therapeutics

July 9, 2007

With support from FRAXA, Seaside Therapeutics developed STX107, an mGluR5 antagonist for Fragile X. Though development ended, it advanced key clinical insights.

Taurine and Somatostatin as Potential Treatments for Fragile X Syndrome: A Unifying Neuro-Endocrine Hypothesis

March 21, 2007

Dr. Abdeslem El Idrissi’s FRAXA-funded study tested somatostatin and taurine in Fragile X mice, revealing taurine’s promise as an accessible therapy.

Jay Gibson, PhD, at University of Texas at Southwestern, FRAXA research grant

Decreased Excitatory Drive onto Parvalbumin-Positive Neocortical Inhibitory Neurons in a Mouse Model of Fragile X Syndrome

February 6, 2007

Drs. Jay Gibson and Kimberly Huber used FRAXA funding to uncover whether disrupted brain inhibition drives Fragile X, paving the way for targeted therapies.

Electrophysiological, Biochemical and Immunohistochemical Characterization of Kv3.1 in Auditory Brainstem Nuclei in the Fragile X Knockout Mouse

January 9, 2007

Dr. Leonard Kaczmarek’s Yale lab revealed how Fragile X disrupts potassium channels, impairing auditory processing and fueling sensory overload.

Baclofen: GABA(B) Receptor Supersensitivity and Normalization of Behavioral Abnormalities by Various GABA(B) Agonists Including Baclofen in FMRP Deficient Mice

October 16, 2006

FRAXA’s $110K grant supported Dr. Miklos Toth at Cornell in discovering that baclofen corrects the heightened startle response in Fragile X mice.

Hypothalamic Pituitary Adrenal (HPA) Axis Dysregulation in Fragile X Syndrome

August 23, 2006

With $62K from FRAXA, Dr. Carolyn Smith studied the HPA stress axis in Fragile X mice, finding hormonal stress recovery unaffected. Results published.

Richard Paylor, PhD, at Baylor College of Medicine, FRAXA Research grant

Therapeutic Interventions in FMR1 Knockout and Transgenic Mice: Role of the FMR1 Gene

February 13, 2006

With FRAXA’s $229K grant, Baylor researchers showed that Fragile X protein levels in the brain affect activity and anxiety in mice and humans.

Joseph LeDoux, PhD, at New York University, FRAXA research grant

Examining the Amygdala in Mouse Models of Fragile X

February 13, 2006

How does the amygdala impact Fragile X? With a $63,000 FRAXA grant, Dr. Joseph LeDoux’s NYU team set out to uncover crucial brain connections.

Social Deficits in Fragile X Syndrome: Do Gene-Gene Interactions Play a Role?

February 13, 2006

FRAXA’s $100K grant supported Drs. Jean Lauder and Sheryl Moy at UNC in exploring gene–gene interactions in Fragile X.

Catherine Choi, Drexel University, FRAXA research grant

Pharmacologic Interventions in the Fmr1 KO Mouse

February 13, 2006

With $48.6K from FRAXA, Dr. Catherine Choi at Drexel studied Fragile X knockout mice to identify potential treatment targets.

Composition and Dynamics of FMRP-Containing RNP Complexes

September 20, 2005

A $30K FRAXA grant helped Dr. Barbara Bardoni at INSERM, France, explore the biochemistry and function of the Fragile X protein.

Clive Svendsen, PhD, at University of Wisconsin, FRAXA research grant

Generating Human Neurons Carrying the Fragile X Mutation

February 26, 2004

FRAXA’s $50K grant supported Dr. Clive Svendsen at Wisconsin in growing Fragile X stem cells to better understand gene characteristics.

Isolating and Characterizing the mRNAs That Bind FMRP

January 14, 2003

With $60K from FRAXA, Dr. Denman’s team investigated how the Fragile X protein FMRP functions in the brain.

Linda Crnic, PhD, at University of Colorado, FRAXA research grant

Treatment of a Mouse Model of Fragile X Syndrome with MPEP

January 1, 2003

A $49K FRAXA grant helped Dr. Linda Crnic’s Colorado team study chronic MPEP use to ease Fragile X symptoms while preserving cognition.

Robert Darnell, MD, PhD, at Rockefeller University, FRAXA research grant

Identification of Specific RNA Targets of FMRP

February 27, 2001

FRAXA-funded Dr. Darnell’s Rockefeller team uncovered how FMRP works and how its absence alters brain function. Published results.

Mina Johnson-Glenberg, PhD, at University of Wisconsin, FRAXA research grant

Neural Network Model of Working Memory in Fragile X Syndrome

February 27, 2000

FRAXA-funded Dr. Johnson-Glenberg explored how Fragile X impacts memory and learning, offering insight into cognitive challenges.

Melatonin Clinical Trial in Fragile X

February 27, 2000

FRAXA-funded Dr. Hagerman’s UC team explored melatonin’s effects in Fragile X, advancing understanding of treatment options. Results published.

Reactivation of the FMR1 Gene in Fragile X Patients Cells in Culture

January 14, 2000

FRAXA-funded Dr. Neri’s team in Italy tested strategies to reactivate the Fragile X gene, advancing curative research.

Glutamate Metabolism in Fragile X Mouse Brain

Using Fenobam to Reduce APP and Abeta in Fragile X Mice

AMPAkines and BDNF in Fragile X: UCI Researchers Restore Memory Process in Fragile X

Targeting the Role of Group 1 Metabotropic Glutamate Receptors

Effects of Alternative Splicing at FMR1 Exon 15 on Understanding Fragile X Syndrome

In Vitro Reactivation of the Fragile X Gene

FRAXA Contributes $10,000 to NIH grant to Seaside Therapeutics

Taurine and Somatostatin as Potential Treatments for Fragile X Syndrome: A Unifying Neuro-Endocrine Hypothesis

Decreased Excitatory Drive onto Parvalbumin-Positive Neocortical Inhibitory Neurons in a Mouse Model of Fragile X Syndrome

Electrophysiological, Biochemical and Immunohistochemical Characterization of Kv3.1 in Auditory Brainstem Nuclei in the Fragile X Knockout Mouse

Baclofen: GABA(B) Receptor Supersensitivity and Normalization of Behavioral Abnormalities by Various GABA(B) Agonists Including Baclofen in FMRP Deficient Mice

Hypothalamic Pituitary Adrenal (HPA) Axis Dysregulation in Fragile X Syndrome

Therapeutic Interventions in FMR1 Knockout and Transgenic Mice: Role of the FMR1 Gene

Examining the Amygdala in Mouse Models of Fragile X

Social Deficits in Fragile X Syndrome: Do Gene-Gene Interactions Play a Role?

Pharmacologic Interventions in the Fmr1 KO Mouse

Composition and Dynamics of FMRP-Containing RNP Complexes

Generating Human Neurons Carrying the Fragile X Mutation

Isolating and Characterizing the mRNAs That Bind FMRP

Treatment of a Mouse Model of Fragile X Syndrome with MPEP

Identification of Specific RNA Targets of FMRP

Neural Network Model of Working Memory in Fragile X Syndrome

Melatonin Clinical Trial in Fragile X

Reactivation of the FMR1 Gene in Fragile X Patients Cells in Culture

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