A renewal for a training grant (T32) for post-doctoral fellows in Genetics and Genomics of Muscle is described. Training faculty is drawn from Children's National Medical Center, NIH intramural program, and George Washington University. Research and training topics focus on understanding muscle tissue in health and disease (dystrophy and damage), with an emphasis on genome-enabled approaches and translational research. Lab bench-based technological training includes state-of-art research methods such as genomics, proteomics, epigenomics, biomarker discovery and validation, drug development and pre-clinical efficacy and toxicity. This technological bench training is heavily augmented with training in bioinformatics and statistics, and appropriate conduct of research. Unique opportunities for trainees include integration into neuromuscular clinical training at both NIH NINDS and CNMC (2/9 previous awardees), ABMG certification (1/9), and training in clinical trials and outcome measures in the CINRG group and NIH intramural program (2/9). Training in private-public partnerships, industry spin-offs, intellectual property development, and regulatory science are also key strengths of the T32. The T32 program is bolstered through synergism by a strong foundation of federally-funded Center and Core grants on muscle translational research led by Training Faculty that provide rich core facilities and pilot funding opportunities for the T32 fellows. These include a Center of Research Translation (NIAMS P50), National Center for Medical Rehabilitation Research (NICHD/NINDS R24), Research in Pediatric Developmental Pharmacology (NICHD U54), CINRG clinical trial network (NIAMS RO1s and DoD grants on outcome measures), Mouse Pathobiology (NIH OD R26), Intellectual and Developmental Disabilities Research Center (NICHD P30), and pediatrics-focused CTSA (NCATS). The initial award filled all available slots through a highly competitive process, with fellows publishing high impact papers, and all graduates retained in independent academic biomedical research positions, including an endowed chair leading a muscular dystrophy program, and head of a molecular diagnostics laboratory. ~30% of trainees were physician/scientists - a stated goal of the original training program. We will train 2 postdoctoral M.D. trainee and 4 postdoctoral Ph.D. trainees in Yr01; 2 postdoctoral M.D. trainees and 5 postdoctoral Ph.D. trainees in Yrs02 through Yr05. A total of 14 postdoctoral trainees will be appointed to this T-32 program over the 5 year funding cycle. At completion, the T32 postdoctoral fellows will be able to independently complete research projects, collaborate in multi-disciplinary research projects, publish research results, apply for independent funding, and successfully interview in academic, pharmaceutical and biomedical research institutions.

Public Health Relevance

Muscle tissue is the largest organ system in the human body, and is the site of common human ailments, such as type 2 diabetes (insulin resistance), muscular dystrophy, frailty in older individuals, and injury. Research on muscle is under-represented in the national research portfolio, and this training grant proposes to continue to train outstanding young physicians and scientists to become the muscle researchers, industry and policy leaders of tomorrow.

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Institutional National Research Service Award (T32)
Project #
5T32AR056993-09
Application #
9481177
Study Section
Special Emphasis Panel (ZAR1)
Program Officer
Boyce, Amanda T
Project Start
2010-05-28
Project End
2020-04-30
Budget Start
2018-05-01
Budget End
2019-04-30
Support Year
9
Fiscal Year
2018
Total Cost
Indirect Cost
Name
Children's Research Institute
Department
Type
DUNS #
143983562
City
Washington
State
DC
Country
United States
Zip Code
20010
Akpulat, Ugur; Wang, Haicui; Becker, Kerstin et al. (2018) Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD. Mol Ther Nucleic Acids 13:534-542
Novak, James S; Jaiswal, Jyoti K; Partridge, Terence A (2018) The macrophage as a Trojan horse for antisense oligonucleotide delivery. Expert Opin Ther Targets 22:463-466
Hubal, Monica J; Nadler, Evan P; Ferrante, Sarah C et al. (2017) Circulating adipocyte-derived exosomal MicroRNAs associated with decreased insulin resistance after gastric bypass. Obesity (Silver Spring) 25:102-110
Novak, James S; Hogarth, Marshall W; Boehler, Jessica F et al. (2017) Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle. Nat Commun 8:941
Boehler, Jessica F; Hogarth, Marshall W; Barberio, Matthew D et al. (2017) Effect of endurance exercise on microRNAs in myositis skeletal muscle-A randomized controlled study. PLoS One 12:e0183292
Echigoya, Yusuke; Lim, Kenji Rowel Q; Trieu, Nhu et al. (2017) Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy. Mol Ther 25:2561-2572
Kahle, Kristopher T; Flores, Bianca; Bharucha-Goebel, Diana et al. (2016) Peripheral motor neuropathy is associated with defective kinase regulation of the KCC3 cotransporter. Sci Signal 9:ra77
Coley, William D; Bogdanik, Laurent; Vila, Maria Candida et al. (2016) Effect of genetic background on the dystrophic phenotype in mdx mice. Hum Mol Genet 25:130-45
Punetha, Jaya; Kesari, Akanchha; Uapinyoying, Prech et al. (2016) Targeted Re-Sequencing Emulsion PCR Panel for Myopathies: Results in 94 Cases. J Neuromuscul Dis 3:209-225
Harris, Elizabeth; Bladen, Catherine L; Mayhew, Anna et al. (2016) The Clinical Outcome Study for dysferlinopathy: An international multicenter study. Neurol Genet 2:e89

Showing the most recent 10 out of 28 publications