The need to train the next generation of medical geneticists has never been more urgent. Our biggest challenge in Medicine is to handle the flood of genomic information associated with new methodologies such as next generation sequencing. The overwhelming power of these new technologies is transforming the traditional approach to medical diagnosis and functional investigations. This renewal training proposal will prepare the future generation to tackle a new reality in which: 1) genetic testing and technology evolves rapidly, 2) most clinical testing is done outside of the medical genetics clinic, either by other specialists or direct-to-consumer, 3) there is a growing understanding of the influence of genetics on a vast array of human disease, and 4) precision medicine will shape future patient care. Our UCLA Intercampus Medical Genetics Post-Doctoral Training Program utilizes the outstanding, diverse faculty and facilities of four campuses: UCLA School of Medicine, Harbor-UCLA Medical Center, Cedars-Sinai Medical Center and Children's Hospital of Orange County. The faculty of this program provides a wealth of research opportunities, varying from basic molecular biology, genomics, cell biology, stem cell research, biochemical genetics, cytogenetics, population genetics, to clinical genetics and dysmorphology. Our collaborative Faculty is organized around three thematic areas: Genomic Medicine, Phenotyping in the Genomic Era, and Treatment of Genetic Disease. We will provide our trainees with a broad knowledge of medical genetics and an intensive experience in genetic research over two years, so that they may enter the academic community as independent investigators and future field leaders. Major strengths of the training environment include a vibrant clinical genetics program with access to an ethnically varied population, state-of-the-art diagnostic laboratories in cytogenetics, biochemical genetics, and molecular genetics, including a clinical genomics center that produces exome sequencing, a wide variety of well-funded research laboratories, a longstanding training experience of faculty, a specific postdoctoral course in medical genetics, an experienced associate director for Diversity to increase the recruitment of underrepresented trainees and a deep institutional commitment. Moreover, we have expanded our faculty and recruitment pool to specialists outside of Medical Genetics, recognizing that most genetics will in fact be practiced and studied beyond the traditional boundaries of our field. While our program has a 30-year history of success, this renewal proposal is focused squarely on the future, and enthusiastically embraces the enormous changes that new discoveries will bring.

Public Health Relevance

Recent advances in genetics and genomics are allowing us to identify the genetic variations that contribute to common as well as rare diseases. There is a rapidly increasing need for expertise in genetics at the clinical, biochemical, cellular, molecular and informatics levels. This training program will attract and train bright young scientists and physicians who will then be able to do meaningful research into the causes, prevention and treatment of genetic diseases, as well as perform and interpret the rapidly evolving clinical and genomic tests necessary to accomplish these goals.

Agency
National Institute of Health (NIH)
Institute
National Institute of General Medical Sciences (NIGMS)
Type
Institutional National Research Service Award (T32)
Project #
2T32GM008243-31
Application #
9209918
Study Section
Special Emphasis Panel (ZGM1)
Program Officer
Sledjeski, Darren D
Project Start
1987-07-01
Project End
2020-06-30
Budget Start
2017-07-01
Budget End
2018-06-30
Support Year
31
Fiscal Year
2017
Total Cost
Indirect Cost
Name
University of California Los Angeles
Department
Genetics
Type
Schools of Medicine
DUNS #
092530369
City
Los Angeles
State
CA
Country
United States
Zip Code
90095
Le, Steven Q; Kan, Shih-Hsin; Clarke, Don et al. (2018) A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I. Mol Ther Methods Clin Dev 8:42-51
Clarke, Don; Pearse, Yewande; Kan, Shih-Hsin et al. (2018) Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice. Mol Ther Methods Clin Dev 10:113-127
Angarita, Stephanie A K; Truong, Brian; Khoja, Suhail et al. (2018) Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice. Mol Genet Metab 124:114-123
Gallant, N M; Leydiker, K; Wilnai, Y et al. (2017) Biochemical characteristics of newborns with carnitine transporter defect identified by newborn screening in California. Mol Genet Metab 122:76-84
Kan, Shih-Hsin; Le, Steven Q; Bui, Quang D et al. (2016) Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behav Brain Res 312:265-71
Wang, Jessica Jen-Chu; Rau, Christoph; Avetisyan, Rozeta et al. (2016) Genetic Dissection of Cardiac Remodeling in an Isoproterenol-Induced Heart Failure Mouse Model. PLoS Genet 12:e1006038
Lee, Patrick C; Truong, Brian; Vega-Crespo, Agustin et al. (2016) Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells. Mol Ther Nucleic Acids 5:e394
Marques, Felipe; Tenney, Jessica; Duran, Ivan et al. (2016) Altered mRNA Splicing, Chondrocyte Gene Expression and Abnormal Skeletal Development due to SF3B4 Mutations in Rodriguez Acrofacial Dysostosis. PLoS Genet 12:e1006307
Tai, Denise S; Hu, Chuhong; Kim, Elizabeth H et al. (2015) Augmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses. Pediatr Res 78:239-246
Rickabaugh, Tammy M; Baxter, Ruth M; Sehl, Mary et al. (2015) Acceleration of age-associated methylation patterns in HIV-1-infected adults. PLoS One 10:e0119201

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