Funding Opportunities

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PI: Rachelle Crosbie-Watson
Professor, Integrative Biology & Physiology, UCLA
Sarcospan Modulators to Treat Duchenne/Becker Muscular Dystrophy

PI: Karen Reue, Ph.D.
Professor, Human Genetics, UCLA
Statin Drug Therapy for Dysferlinopathy

PI: Deirdre Scripture-Adams, Ph.D.
Research Biologist, UCLA
Time of Flight Mass Cytometry Based on Immune Biomarker

PI: Reza Ardehali, M.D., Ph.D.
Associate Professor of Cardiology, UCLA
The Role of Cardiac Pericytes in the Development and Progression of DMD Cardiomyopathy

PI: Marino DiFranco, Ph.D.
Adjunct Professor and Research Physiologist, Department of Physiology, UCLA
Mechanical Assessment of the Efficacy of Gene Therapy Approaches to Restore Function in Dystrophin-Deficient Muscles

PI: Jonathan Wanagat, M.D., Ph.D.
Assistant Professor of Medicine, Division of Geriatrics, UCLA
Quantitative Imaging of the Dystrophin-Glycoprotein Complex

PI: Frank Petrigliano, M.D.
Professor of Orthopedic Surgery, UCLA
Defining the Myogenic Niche for Rotator Cuff Skeletal Muscle Regeneration by Administration of hPSC-pericytes

PI: Daniel Ennis, Ph.D.
Professor of Radiological Sciences and Bioengineering, UCLA
Measuring Cardiomyocyte Performance In Vivo with MRI

PI: Melissa Spencer, Ph.D.
Professor of Neurology, UCLA
Generation of a Humanized Mouse Model of DMD

PI: Huan Meng, Ph.D.
Assistant Professor of Medicine, UCLA
Custom Design of Polyrotaxane Nanocarrier for CRISPR/Cas9 Delivery in DMD

PI: Stanley F. Nelson, M.D.
Professor of Human Genetics, UCLA
Assessment of Vacuum Needle Biopsy System for Sampling in Muscle Disease

PI: Shantanu Joshi, Ph.D.
Department of Neurology, UCLA
Brain Imaging Signatures in Duchenne Muscular Dystrophy and their Correlates with Cognitive Function

PI: Sandra Loo, Ph.D.
Department of Psychiatry and Biobehavioral Sciences, UCLA
Behavioral, Cognitive and Neuropsychological Phenotypes in Duchenne Muscular Dystrophy

PI: Atsushi Nakano, Ph.D., UCLA Molecular
Cellular and Developmental Biology, UCLA
Developing iPSC-Derived Cardiomyocyte as a Model System for Evaluating Therapeutic Intervention of DMD Cardiomyopathy

This year the Pilot and Feasibility Seed Grant Program received many excellent quality applications. We are pleased to be able to provide funding to those that are highly relevant to the missions of the P30 and CDMD.

PI: M. Carrie Miceli, Ph.D.               
Professor, Microbiology, Immunology & Molecular Genetics, UCLA               
Co-PIs: Dr. Stanley Nelson, Dr. April Pyle, Dr. Rachelle Crosbie-Watson, Dr. Linda Baum, and Dr. Melissa Spencer
Stem Cell Approaches for Combination DMD Exon Skipping Therapy

PI: Karen Reue, Ph.D.               
Professor, Human Genetics, UCLA               
Co-PI: Andrea Hevener
Mechanisms of Muscle Metabolism and Autophagy in Muscular Dystrophy and Statin Myopathy

PI: Rachelle Crosbie-Watson, Ph.D.               
Professor, Integrative Biology and Physiology Neurology, UCLA               
Co-PI: Dr. Kenneth Roos
Restoration of Muscle Cell Adhesion to Treat Cardiomyopathy in Muscular Dystrophy

PI: Brigitte Gomperts, M.D.               
Assistant Professor, Pediatric Hematology/Oncology, UCLA               
Co-PI: Dr. April Pyle
Improving Our Understanding of Duchenne Muscular Dystrophy by Modeling the Muscle Cell Niche

Loren Fong, Ph.D.               
Professor of Cardiology, David Geffen School of Medicine, UCLA
A Fresh Strategy for Treating Muscular Dystrophies Caused by Mutations in LMNA

Dr. Nancy Halnon    
Nancy Halnon, M.D.               
Associate Professor of Pediatrics and Pediatric Cardiology, David Geffen School of Medicine, UCLA
Evaluating Cardiopulmonary Exercise Testing for Duchenne Muscular Dystrophy

Dr. Leanne Jones     
Leanne Jones, Ph.D.               
Associate Professor of Molecular, Cell and Developmental Biology, UCLA
Directing Mitochondrial Biogenesis to Enhance Stem Cell Maintenance and Transplantation

Dr. Jonathan Wanagat    
Jonathan Wanagat, M.D., Ph.D.               
Assistant Professor of Medicine, Division of Geriatrics at UCLA
Multiplex Protein Quantitation of Single Muscle Fiber Sections

Dr. Holden Wu     
Holden Wu, Ph.D.               
Adjunct Assistant Professor of Radiology at UCLA
Development and Evaluation of Free-Breathing High-Resolution 3D MRI for Characterization of Fibro-Fatty Replacement of Cardiac Muscle in Patients with Duchenne Muscular Dystrophy

In 2012, the Pilot and Feasibility Seed Grant Program received many excellent applications of high quality. The Center for Duchenne Muscular Dystrophy at UCLA was able to partner with the P30 Core Center to provide matching funds for applications aligned with the overall mission of the CDMD, thus extending the number of awards funded this year!

*Daniel Cruz, M.D., Ph.D.
Assistant Professor of Cardiology, David Geffen School of Medicine, UCLA
Harnessing the Microenvironment to Instruct Innate Immune Responses in Duchenne Muscular Dystrophy

Dr. Linda Baum     
*Linda Baum, M.D., Ph.D.               
Professor of Pathology and Laboratory Medicine, David Geffen School of Medicine, UCLA
Characterization of the Human Skeletal Muscle Cell Glycome and Identification of Glycan-related Functions in Membrane Stabilization and Extracellular Matrix Interactions

Dr. Gail Thomas
*Gail Thomas, Ph.D.
Research Scientist III of The Heart Institute, Cedars-Sinai Medical Center
Screening of NO-Donating Compounds to Alleviate Functional Muscle Ischemia in mdx Mice

Dr. Daniel Ennis     
*Daniel Ennis, Ph.D.               
Assistant Professor of Radiology, UCLA
Establishment of Quantitative Cardiac Magnetic Resonance Imaging as a Clinical End Point for Evaluating Ventricular Function in Patients with Duchenne Muscular Dystrophy

Dr. Yibin Wang     
Yibin Wang, Ph.D.               
Professor of Anesthesiology, Physiology and Medicine, UCLA
Fox1 Mediated Transcriptome Remodeling in Muscle Development and Diseases

Dr. Ming Guo    
Ming Guo, M.D., Ph.D.               
Associate Professor of Neurology, Molecular and Medical Pharmacology, UCLA
Mitochondrial DNA Quality Control in Drosophila and Humans

(* Indicates CDMD supplement)

PI: April Pyle, Ph.D.
UCLA Department of Microbiology, Immunology and Molecular Genetics, UCLA
Development of iPSC Cells as Pre-Clinical Screening Tools for DMD

PI: Eileen Fowler, Ph.D.
UCLA Department of Orthopaedic Surgery
Outcome Measures for Clinical Trials in DMD

PI: Melissa Spencer, Ph.D.
UCLA Department of Neurology
Investigation of CaMK signaling in LGMD2A

Principal Investigator:
Rachelle H. Crosbie, Ph.D., UCLA Department of Integrative Biology and Physiology 

Co-Investigator:
Linda G. Baum, M.D., Ph.D., UCLA Department of Pathology and Laboratory Medicine
Novel Mechanisms to Enhance Utrophin Expression and Muscle Cell Function

Principal Investigator:
Douglas L. Black, Ph.D.
UCLA Department of Microbiology, Immunology and Molecular Genetics
Howard Hughes Medical Institute

Co-Investigator:
Peter Stoilov, Ph.D.
UCLA Department of Microbiology, Immunology and Molecular Genetics
Screening of Small Molecules Targeting the Splicing of SMN2 Exon 7 to Identify
Lead Compounds for Treatment of Spinal Muscular Atrophy

Principal Investigator:
Stanley Nelson, M.D.
UCLA Departments of Human Genetics and Psychiatry
Implementation of Small Molecule High Throughput Screens for Fibrosis Inhibition