P50HD105351

Boston Children's Hospital/HMS Intellectual and Developmental Disabilities Research Center - Abstract

Our overarching vision of the Boston Children's Hospital and Harvard Medical School Intellectual and Developmental Disorders Research Center (IDDRC) is to improve the lives of individuals with IDD with timely and efficient translation of scientific research through collaboration among our institutions' exceptional investigators and clinicians in partnership with the external IDD community.

To achieve our broad vision, we organize the center's research around four clearly defined themes:

1) Discovery of genetic and non-genetic causes of IDD;
2) Determination of the cellular bases of IDDs using advanced imaging and analysis tools;
3) Identification of translational phenotypes in animal models of IDD to validate therapeutics;
4) Accelerated translation of research discoveries into new prevention and treatment strategies for IDDs.

The center currently supports 106 research projects and 68 investigators through the Administrative Core and the four Scientific Cores.

The Administrative Core is the hub of the center as it provides both scientific and administrative leadership. It promotes synergistic, interdisciplinary interactions that address IDD-related issues at multiple levels, trains the next generation of young investigators, and facilitates outreach and dissemination of IDD research to diverse audiences.

The Genetic Analysis and Editing Core (GAEC) provides access to the latest technological advances both in genetic analysis and in gene editing.

The Cellular Imaging Core (CIC) facilitates the study of cellular and circuit biology through state-of-the-art imaging and image analysis services. These services enable visualization of fixed tissue, in vitro organ explants, and in vivo awake behaving model organisms.

The Animal Behavior and Physiology (AB&P) Core provides investigators with access to a wide range of validated technologies and scientific expertise for in vivo rodent behavioral, biochemical, and physiological measures in a well-controlled and rigorous preclinical setting.

Finally, the Clinical Translational Core (CTC) provides full access for IDDRC PIs to all services required for the translation of research discoveries into clinical innovation. This includes biosample collection and storage, generation of patient-derived stem cell models of diseases, drug screening platforms on the preclinical side, and neurobehavioral and electrophysiological assessment of IDD patients of different age groups. The CTC also offers statistical and regulatory support for clinical trials.

The cores all interact through shared projects, providing complementary expertise and tools to address unique aspects of central scientific questions. The core directors meet to exchange ideas and optimize resource utilization in the monthly Executive Committee meetings.

This integrated approach aims to enhance the translational potential of basic research in IDD by putting patients at the center of the drug discovery cycle, starting with genetic and molecular screens through clinical trials. The center engages frequently with patient advocacy groups and the IDD community in a bi-directional manner to ensure that their needs and concerns steer the center's efforts.

Children's Hospital Corporation

TO CONDUCT AND SUPPORT LABORATORY RESEARCH, CLINICAL TRIALS, AND STUDIES WITH PEOPLE THAT EXPLORE HEALTH PROCESSES. NICHD RESEARCHERS EXAMINE GROWTH AND DEVELOPMENT, BIOLOGIC AND REPRODUCTIVE FUNCTIONS, BEHAVIOR PATTERNS, AND POPULATION DYNAMICS TO PROTECT AND MAINTAIN THE HEALTH OF ALL PEOPLE. TO EXAMINE THE IMPACT OF DISABILITIES, DISEASES, AND DEFECTS ON THE LIVES OF INDIVIDUALS. WITH THIS INFORMATION, THE NICHD HOPES TO RESTORE, INCREASE, AND MAXIMIZE THE CAPABILITIES OF PEOPLE AFFECTED BY DISEASE AND INJURY. TO SPONSOR TRAINING PROGRAMS FOR SCIENTISTS, DOCTORS, AND RESEARCHERS TO ENSURE THAT NICHD RESEARCH CAN CONTINUE. BY TRAINING THESE PROFESSIONALS IN THE LATEST RESEARCH METHODS AND TECHNOLOGIES, THE NICHD WILL BE ABLE TO CONDUCT ITS RESEARCH AND MAKE HEALTH RESEARCH PROGRESS UNTIL ALL CHILDREN, ADULTS, FAMILIES, AND POPULATIONS ENJOY GOOD HEALTH. THE MISSION OF THE NICHD IS TO ENSURE THAT EVERY PERSON IS BORN HEALTHY AND WANTED, THAT WOMEN SUFFER NO HARMFUL EFFECTS FROM REPRODUCTIVE PROCESSES, AND THAT ALL CHILDREN HAVE THE CHANCE TO ACHIEVE THEIR FULL POTENTIAL FOR HEALTHY AND PRODUCTIVE LIVES, FREE FROM DISEASE OR DISABILITY, AND TO ENSURE THE HEALTH, PRODUCTIVITY, INDEPENDENCE, AND WELL-BEING OF ALL PEOPLE THROUGH OPTIMAL REHABILITATION.

93.865 - Child Health and Human Development Extramural Research

National Institute of Child Health and Human Development (NICHD) [HHS - NIH]

Boston, Massachusetts 021155724 United States

Single Zip Code

Intellectual and Developmental Disabilities Reserch Centers [IDDRC] FY 2021 (P50 Clinical Trial Optional) (RFA-HD-21-009)

Amendment Since initial award the total obligations have increased 427% from $1,326,000 to $6,990,000.