Grant – Winter 2013 – FSHD - Michael Kyba, Ph.D.
Michael Kyba, associate professor of pediatrics at the University of Minnesota in Minneapolis, was awarded an MDA research grant totaling $358,227 over a period of three years to study muscle developmental deficits in facioscapulohumeral muscular dystrophy (FSHD). The new grant complements previous MDA-funded work by Kyba to identify and test experimental therapies in FSHD.
FSHD is caused by aberrant expression (activity) of a gene called DUX4, which is usually silenced in adult muscle tissue. DUX4 turns on other genes, and activity of these genes is believed to cause disease by multiple mechanisms, including direct toxicity, interference with normal cell function and provoking the immune system.
“The mechanism of muscle degeneration in FSHD is enigmatic,” Kyba says, since unlike other muscular dystrophies, there is apparently neither ongoing muscle fiber damage, nor ongoing regeneration. “The mechanism of how genetic changes actually lead to deteriorated muscle is still very unclear.”
To answer some of those questions, he has generated muscle cells from patient skin cells in order to study the effects of the mutation that causes FSHD. He will now use newly developed “gene-editing” technology to correct the FSHD mutation, and to compare muscle development between uncorrected and corrected cells.
“This project will shed light on the molecular mechanism underlying FSHD, and will provide tools for genetic correction of the FSHD mutation in cells in culture,” says Kyba. “Understanding the genetic mechanism is essential to developing rational pharmaceutical therapies for FSHD. In addition, with a view toward future cell therapies, having methods to correct the genetic mutation that causes FSHD will facilitate autologous (self) transplantation of genetically corrected cells.”
Funding for this MDA grant began Feb. 1, 2013.
Grantee: FSHD - Michael Kyba, Ph.D.
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Grant - Spring 2014 - MM - David Chan, M.D., Ph.D.
David Chan, a professor of biology at the California Institute of Technology in Pasadena, has been awarded an MDA research grant totaling $253,800 over three years, to study mitochondria in mice. Mitochondria are the energy-producing parts of cells, and an aspect of their normal functioning involves fusing together and dividing. Chan and colleagues will study mice with defects in the fusion or division of mitochondria to improve the understanding of mitochondrial myopathies.
Funding for this MDA research grant began May 1, 2014.
Grantee: MM - David Chan, M.D., Ph.D.
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Grant - Summer 2012 - CMT — Stephan Züchner, M.D.
Stephen Züchner, associate professor of human genetics and neurology at the University of Miami Miller School of Medicine in Florida, was awarded an MDA research grant totaling $390,000 over three years to identify genes responsible for Charcot-Marie-Tooth disease (CMT).
"Genetic research over the past 25 years has identified more than 50 different CMT genes,” Züchner explains. “While the subtype CMT1 is largely explained by these genes, CMT2 and a number of other subtypes of CMT are less well-understood.”
It’s thought that many more genes need to be found to explain the majority of CMT found in people with the disease, but a new generation of DNA sequencing machines — called next-generation sequencers — is making that endeavor much easier and cheaper
“We have been pioneering the application of these new instruments for neuromuscular and other diseases,” Züchner says, “and have already identified and published multiple novel genes using this approach in a number of diseases.”
In his new work, Züchner and colleagues are focused on identifying additional genes that cause CMT.
“We believe that the more genes we know, the better will be our understanding of gene networks that work together in a process that leads to disease,” Züchner says. “Instead of focusing on a single gene, there will be benefits in understanding the entire disease process.”
Such understanding may lead to development of therapies that, instead of targeting a specific gene, influence gene networks or pathways — meaning a single treatment could help people with mutations in different genes.
Funding for this MDA grant began Aug. 1, 2012.
Grantee: CMT — Stephan Züchner, M.D.
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Grant – Winter 2013 – FA - Mark Payne, M.D.
Mark Payne, professor of pediatrics and medical and molecular genetics at the Indiana University School of Medicine in Indianapolis, was awarded an MDA research grant totaling $298,048 over a period of two years to study ways to overcome the vulnerability of heart muscle in Friedreich’s ataxia (FA).
Heart muscle is especially vulnerable in FA, leading to the possibility of severe heart failure and early death. The reason, Payne says, appears to relate to how mitochondria, the cell’s energy producers, obtain their own cellular fuel. “It appears that the mitochondria in the heart, which normally produce energy for the heart to beat, are not capable of using certain fuels (fats and sugars) to make energy. The result is that the heart does not have enough energy to withstand stressful situations.”
Payne will study this phenomenon further in a mouse model of FA, to understand altered mitochondrial function in the heart that may contribute to the development of heart failure. “In particular, we will determine if the mitochondria are capable of using fats and sugars from the diet in an appropriate manner to make energy,” he says, using a variety of advanced molecular biology and imaging techniques. He also will study whether supplying a good copy of the mutated gene responsible for the disease can reverse the faulty metabolism of the heart.
“The goal of this project is to understand the basic mechanisms of heart failure in this disease, and then develop approaches to improve heart function and save lives,” Payne says.
Funding for this MDA grant began Feb. 1, 2013.
Grantee: FA - Mark Payne, M.D.
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Grant - Spring 2014 - MM - Catarina Quinzii, M.D
Catarina Quinzii, an assistant professor at Columbia University Medical Center in New York, has been awarded an MDA research grant totaling $253,800 over three years to investigate how flaws in the gene for a protein called RMND1 lead to abnormalities in mitochondria, the energy-producing parts of cells. Quinzii will conduct experiments in human cells, mouse cells and mice with abnormal or deficient RMND1 protein, with an eye to developing a treatment for a human mitochondrial myopathy that results from an RMND1 gene mutation.
Funding for this MDA research grant began May 1, 2014.
Grantee: MM - Catarina Quinzii, M.D
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Grant - Summer 2012 - CMT — Bogdan Beirowski, M.D., Ph.D.
Postdoctoral research scholar Bogdan Beirowski, in the department of genetics at the Washington University School of Medicine in St. Louis, was awarded an MDA development grant totaling $180,000 over three years to study how defective Schwann cells lead to nerve-cell damage in Charcot-Marie-Tooth disease (CMT).
MDA development grants are awarded to exceptional postdoctoral candidates who have the best chance of becoming independent researchers and future leaders of neuromuscular disease research.
Degeneration of axons — fibers that carry electrical signals between the brain and spinal cord and the rest of the body — is a hallmark of CMT and some forms of Dejerine-Sottas disease, and is thought to be responsible for muscle weakness associated with these diseases.
In some forms of CMT, the primary molecular defect localizes to Schwann cells, which normally help nourish and protect axons by wrapping them in a multilayered membrane called myelin. But it's poorly understood how Schwann cell dysfunction causes axon damage.
Data from previous work suggests that removal of defective myelin (demyelination) causes inflammation that results in axon damage, but Beirowski hypothesizes that "defective Schwann cells deprive long axons of metabolic support, thus contributing to axon degeneration."
Beirowski has developed mouse models in which key metabolic regulators are blocked exclusively in Schwann cells, which he now will use to test whether failure of the cells to deliver nutrients to axons could explain axon loss.
The identification of specific metabolic defects in Schwann cells could lead to the development of CMT therapeutic strategies based on supporting the stability of axons.
Funding for this MDA grant began Aug. 1, 2012.
Grantee: CMT — Bogdan Beirowski, M.D., Ph.D.
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Grant - Spring 2014 -MM - Carlos Moraes, Ph.D.
Carlos Moraes, a professor of neurology, cell biology and anatomy at the University of Miami (Fla.), has been awarded an MDA research grant totaling $253,800 over three years to study a potential treatment for mitochondrial myopathies, conducting experiments in human cells and in mice. Mitochondria, the energy-producing parts of cells, contain their own DNA, which can develop mutations (flaws) and cause disease. Moraes and colleagues will study DNA-cutting enzymes targeted to mitochondria, hoping to develop this technology to correct disease-causing defects in mitochondrial DNA in patients.
Funding for this MDA research grant began May 1, 2014.
Grantee: MM - Carlos Moraes, Ph.D.
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Grant - Summer 2012 - CMS — Paul Brehm, Ph.D.
Paul Brehm, senior scientist at Oregon Health Science University in Portland, was awarded an MDA research grant totaling $351,648 over three years to study the underlying mechanisms of movement disorders in some forms of congenital myasthenic syndromes (CMS).
Brehm’s research team has identified zebrafish research models that carry mutations in the same proteins that are known to cause some forms of CMS in humans. In both fish and humans, these proteins are localized to the neuromuscular junction (the space where signals are transferred between nerve and muscle). Their deficiency results in movement disorders such as use-dependent fatigue (the inability to perform repetitive tasks).
In one model, known as the rapsyn-deficient line (in which the fish have mutations in the gene for the rapsyn protein), Brehm and his team have been exploring the basis of use-dependent fatigue.
Rapsyn deficiency has been associated with problems on the muscle side of the neuromuscular junction. Brehm’s team has found that the absence of an as-yet unidentified signal produced by muscle regulates the motor neuron and that, ultimately, the absence of this signal appears to be accountable for use-dependent fatigue.
Brehm’s team is working to uncover the mechanism underlying the reduced motor neuron activity that precedes use-dependent fatigue, as well as ways in which to rescue the nerve defect.
“The means to study neuromuscular function using the zebrafish system has been unparalleled among the vertebrates,” Brehm says. “The discovery of zebrafish models that carry these mutant proteins now opens the way to addressing directly the consequences in humans.”
Funding for this MDA grant began Aug. 1, 2012.
Grantee: CMS — Paul Brehm, Ph.D.
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Grant - Spring 2014 - LEMS - Stephen Meriney, Ph.D.
Stephen Meriney, a professor of neuroscience and psychiatry at the University of Pittsburgh, will conduct experiments in cells and mice to test a drug that may be beneficial in Lambert-Eaton myasthenic syndrome (LEMS).
Funding for this MDA research grant began May 1, 2014.
Grantee: LEMS - Stephen Meriney, Ph.D.
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Grant - Summer 2012 - CMD — Madhuri Hegde, Ph.D.
Madhuri Hegde, associate professor and scientific director at Emory Genetics Laboratory, Emory University in Atlanta, was awarded an MDA research grant totaling $262,928 over two years to identify genes that, when mutated, cause congenital muscular dystrophy (CMD).
A significant number of CMD-associated genes have been identified, but in approximately 30 to 40 percent of occurrences of the disease the causative gene remains unknown.
“As understanding of disease mechanisms and the function of the genes involved in the pathology of CMD grows, new cutting-edge therapies may be developed for the cases in which causative genes are currently unknown,” Hedge says.
Hegde and colleagues are working to identify more genes associated with CMD, using a variety of both traditional and next-generation methods designed to determine a person’s entire genetic makeup, such as whole-genome genotyping and whole and targeted next-generation genomic sequencing.
The team also is focusing on understanding the genotype-phenotyperelationship (the correlation of clinical differences with particular mutations) and developing a map of the CMD muscle genome (complete set of DNA),transcriptomes (the set of all molecules of RNA, a chemical cousin to DNA), and proteosomes (protein complexes).
“Next generation sequencing has revolutionized our ability to find new disease-causing genes, but it has not yet been widely applied to neuromuscular diseases,” Hedge says. “Research on new therapies is moving at a rapid pace, so it is important to identify the molecular defect in patients.”
Funding for this MDA grant began Aug. 1, 2012.
Grantee: CMD — Madhuri Hegde, Ph.D.
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Grant - Spring 2014 - EDMD - Howard Worman, M.D.
Howard Worman, a professor of medicine, pathology and cell biology at Columbia University in New York, has been awarded an MDA research grant totaling $253,800 over three years to study genetically modified mice that lack two proteins — emerin and LAP1 — and develop a disorder mimicking the X-linked form of Emery-Dreifuss muscular dystrophy (EDMD). He will study disease progression in these mice and test a potential new treatment in them.
Funding for this MDA research grant began May 1, 2014.
Grantee: EDMD - Howard Worman, M.D.
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Grant - Spring 2014 - DMD - Terence Partridge, Ph.D.
Terence Partridge, a professor at Children’s Research Institute at Children’s National Medical Center in Washington, D.C., was awarded an MDA research grant totaling $253,800 over three years to develop and test a new type of compound that causes exon skipping – a way for cells to make functional proteins from flawed genes -- as a potential treatment for Duchenne muscular dystrophy (DMD). Current exon-skipping drugs for DMD have limited, if any, ability to enter heart muscle cells. Partridge is studying an exon-skipping compound that appears to enter the heart in addition to skeletal muscles and will test it in mice with a DMD-like disorder.
Funding for this MDA research grant began May 1, 2014.
Grantee: DMD - Terence Partridge, Ph.D.
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Grant - Spring 2014 - DMD - Nicoletta Filigheddu, Ph.D.
Nicoletta Filigheddu, a molecular biologist at the University of Piemonte Orientale Amedeo Avogadro in Italy, has been awarded an MDA research grant totaling $253,800 over three years to investigate whether a hormone called ghrelin can improve muscle regeneration or the success of muscle stem cell transplantation in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).
Funding for this MDA research grant began May 1, 2014.
Grantee: DMD - Nicoletta Filigheddu, Ph.D.
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Grant - Spring 2014 - DMD - Federica Montanaro, Ph.D.
Federica Montanaro, Ph.D., a principal investigator at the Center for Gene Therapy at Nationwide Children's Hospital in Columbus, Ohio, and an assistant professor of pediatrics at Ohio State University, was awarded an MDA research grant totaling $253,800 over three years to study how decreased activity in a biochemical signaling pathway called "hedgehog" affects muscle stem cells during muscle repair. She will find out whether increasing hedgehog signaling in mice with a DMD-like disorder preserves muscle function.
Funding for this MDA research grant began May 1, 2014.
Grantee: DMD - Federica Montanaro, Ph.D.
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Grant - Spring 2014 - DMD - Erica Reeves, Ph.D.
Erica Reeves, a biologist and vice president of research and operations at ReveraGen BioPharma, was awarded an MDA translational research grant totaling of $1,015,200 over one year to conduct the first human trial of VBP15, an experimental drug in development to treat Duchenne muscular dystrophy (DMD). This first trial will be in healthy volunteers. VBP15 is designed to combat inflammation and improve membrane stability in DMD-affected muscles and to provide the benefits of corticosteroid drugs like prednisone without or with reduced side effects. The grant was awarded through MDA's Venture Philanthropy (MVP) program.
Funding for this MDA translational research grant began June 2, 2014.
Grantee: DMD - Erica Reeves, Ph.D.
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Grant - Spring 2014 - DMD - Dongsheng Duan, Ph.D.
Dongsheng Duan, a professor in the Department of Molecular Microbiology & Immunology at the University of Missouri in Columbia, has received an MDA research infrastructure grant totaling $169,200 over two years to develop and maintain a national canine tissue bank so that investigators studying Duchenne muscular dystrophy (DMD) will have access to normal and dystrophin-deficient tissue samples taken from dogs. Dystrophin is the muscle protein that is missing in DMD patients. Successful establishment of this tissue bank is expected to accelerate DMD research in numerous directions.
Funding for this MDA research grant began May 1, 2014.
Grantee: DMD - Dongsheng Duan, Ph.D.
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Grant - Spring 2014 - DMD - Darek Gorecki, Ph.D.
Darek Gorecki, a professor of molecular medicine at the University of Portsmouth in the United Kingdom, has been awarded an MDA research grant totaling $253,800 over three years to study whether blocking a specific receptor (molecular docking site) that enhances inflammation can be beneficial to muscle fibers affected by Duchenne muscular dystrophy (DMD). He will conduct experiments in mice.
Funding for this MDA research grant began May 1, 2014.
Grantee: DMD - Darek Gorecki, Ph.D.
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Grant - Spring 2014 - DMD - Da-Zhi Wang, Ph.D.
Da-Zhi Wang, an associate professor of pediatrics at Boston Children's Hospital and Harvard Medical School, was awarded an MDA research grant totaling $253,800 over three years to study the role of a compound called miR-155, a "microRNA," in muscle function and regeneration and to see whether reducing it could help treat Duchenne muscular dystrophy (DMD). Wang has found that mice with lacking miR-155 have better muscle function and regeneration than mice that have this substance. He and his colleagues will study mice with a disorder resembling human muscular dystrophy to define the function of miR-155, with an eye to therapy development targeting this compound.
Funding for this MDA research grant began May 1, 2014.
Grantee: DMD - Da-Zhi Wang, Ph.D.
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Grant - Spring 2014 - CMT/DSD - Kelly Monk, Ph.D.
Kelly Monk, an assistant professor of developmental biology at Washington University in St. Louis, was awarded an MDA research grant totaling $253,800 over three years to investigate a possible therapeutic avenue for treating Charcot-Marie-Tooth disease (CMT) and the related Dejerine-Sottas disease (DSD). Monk and her colleagues have previously found that a protein called GPR126 is required for proper formation and maintenance of the myelin sheath that insulates fibers in the peripheral nervous system, and they will now study how this protein controls this process, which is known as myelination. They will also look at the role of a related protein called GPR56 and will investigate whether either or both of these proteins could represent new drug development targets for CMT or DSD.
Funding for this MDA grant began May 1, 2014.
Grantee: CMT/DSD - Kelly Monk, Ph.D.
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Grant - Summer 2013 - DMD — Hansell Stedman, M.D.
Hansell Stedman, associate professor of surgery at the University of Pennsylvania in Philadelphia, was awarded an MDA research grant totaling $300,000 over a period of three years to study early immune responses during administration of gene therapy.
Gene therapy is a promising treatment strategy for Duchenne muscular dystrophy (DMD) and other muscle diseases, but it has been hindered by the body’s immune response. Genes can be delivered via virus-like particles (viral vectors), but the immune system can identify them as foreign, and mount a response that can inactivate the vector and cause inflammation in the muscle. Stedman plans to study the very earliest phases of the immune response, combining approaches gleaned from immunology, surgical critical care, genetics and virology.
“This mechanistic information will identify rational targets for transient immunosuppression prior to vector administration,” Stedman says, “thereby improving the chances for safe and durable therapy for these devastating childhood-onset diseases.” His work will be performed in the mouse model of DMD.
“In our view, the entire field is tantalizingly close to the development of gene therapy for Duchenne and other forms of muscular dystrophy, but we must explore a number of strategies to address the remaining obstacles,” he says. “Understanding how the innate immune system recognizes injured cells should improve our chances for a successful intervention.”
Funding for this MDA grant began August 1, 2013.
Grantee: DMD — Hansell Stedman, M.D.
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Grant - Winter 2012 - ALS - Terry Heiman-Patterson, M.D.
MDA awarded a research grant totaling $330,000 over a period of three years to Terry Heiman-Patterson, section chief of neuromuscular disorders at Drexel University College of Medicine in Philadelphia. Heiman-Patterson also is medical director of the MDA/ALS Center of Hope at that institution.
The newly awarded funds will help support Heiman-Patterson's work to identify "modifier" genes in mouse models of amyotrophic lateral sclerosis (ALS).
A great deal is known about one of the most commonly used mouse models of ALS — the SOD1 mouse. This model carries mutations in the SOD1 gene and develops some of the same signs and symptoms exhibited by people with SOD1-related ALS.
"Our lab, along with others, have shown that disease severity in these transgenic mice depends on their genetic background," Heiman-Patterson said.
With colleagues, Heiman-Patterson plans to compare a number of mouse models and identify genes that are able to modify disease.
It's already suspected that a region on chromosome 17 modifies disease severity in mice with certain backgrounds. Heiman-Patterson's team intends to validate that the region of chromosome 17 does modify disease, to identify the responsible gene within the region, and to test whether the gene also can affect severity in other models of motor neuron disease.
The identification of modifier genes will provide new targets for therapy development.
Grantee: ALS - Terry Heiman-Patterson, M.D.
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Grant - Winter 2012 - ALS - Kenneth Hensley, Ph.D.
MDA awarded a research grant totaling $328,153 over a period of three years to Kenneth Hensley, associate professor in the departments of pathology and neuroscience, and research director in the department of pathology at the University of Toledo Medical Center in Ohio. The funds will help support Hensley’s study of a potential new target and treatment strategy for amyotrophic lateral sclerosis (ALS).
Recent findings from Hensley’s laboratory and from other groups, Hensley said, suggest that ALS actually begins near the junction of nerve and muscle (the neuromuscular junction, or NMJ).
“We hypothesize that molecules called semaphorins acting inappropriately near the NMJ signal axons, the long fibers that extend and carry information away from the bodies of motor neurons (nerve cells). The semaphorins’ signals cause the axons to move away from the muscle and ‘collapse’ backward toward the spinal cord.”
Research conducted by Hensley and colleagues implicates a protein calledcollapsing response mediator protein-2, or CRMP2, in this process of axon degeneration. The group will now test three distinct pharmacological (drug-based) approaches to interrupting CRMP2-dependent axon degeneration in the SOD1 research mouse model of ALS.
“We have invented and patented small molecule compounds calledlanthionines that bind CRMP2 and inhibit or reverse CRMP2-dependent axonal degeneration,” Hensley said.
The investigators also are researching antibodies that could be administered to people with ALS, where they would block semaphorin binding to neural receptors and prevent the inappropriate activation of CRMP2 pathways.
Grantee: ALS - Kenneth Hensley, Ph.D.
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Grant - Spring 2014 - CMT - Nivedita Jerath, M.D.
Nivedita Jerath, a clinical fellow in neuromuscular medicine at the University of Iowa, has been awarded an MDA clinical research training grant to study driving ability in patients with the type 1A form of Charcot-Marie-Tooth disease (CMT). Because of difficulties with strength and balance, as well as foot abnormalities and tight ankles, the disease may affect driving, which requires quick responses, such as slamming on the brakes or turning the steering wheel quickly. Using a driving simulator as well as a special car that can videotape driver performance on the road, Jerath will investigate whether or not patients with CMT1A can drive normally. If they are found to have driving difficulties, Jerath says, she hopes the findings can be used to develop devices that might help people with CMT1A to drive more safely.
Funding for this MDA grant began July 1, 2014
Grantee: CMT - Nivedita Jerath, M.D.
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Grant - Winter 2012 - ALS - Eric Huang, M.D., Ph.D.
MDA awarded a grant totaling $412,500 over a period of three years to Eric Huang, professor of neuropathology at the University of California in San Francisco. The funds will help support Huang’s work to create cellular and mouse models of amyotrophic lateral sclerosis (ALS) that is caused by mutations in the fused in sarcoma (FUS) gene.
Genetic data have shown that mutations in the FUS gene can be identified in more than 5 percent of people with familial (inherited) ALS.
Huang and colleagues hypothesize that these FUS mutations interfere with the normal production of proteins, ultimately leading to the degeneration and death of the nerve cells called motor neurons.
The team’s goal is to create cellular and transgenic mouse models of FUS-related ALS that can be used to determine the ways in which mutant FUS proteins cause motor neuron degeneration.
Studies in the new FUS transgenic mice are expected to provide novel insights into the disease process and therapeutic targets for ALS, Huang said.
Grantee: ALS - Eric Huang, M.D., Ph.D.
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Grant - Spring 2014 - CMT - Michael Shy, M.D.
Michael Shy, a professor of neurology and pediatrics at the University of Iowa, has been awarded an MDA research grant totaling $253,800 over three years to develop a clinical trial to test a treatment for patients with the type 1B form of Charcot-Marie-Tooth disease (CMT). Based on previous MDA-supported work in mice with a CMT1B-like disorder, Shy and colleagues believe that treating a cellular phenomenon called the ER stress response may help CMT1B patients.
Funding for this MDA grant began May 1, 2014.
Grantee: CMT - Michael Shy, M.D.
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