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Translational and clinical studies targeting y-globin modulation

针对 y 珠蛋白调节的转化和临床研究

基本信息

批准号：
8866459
负责人：
DAVID A WILLIAMS
金额：
$ 146.12万
依托单位：
BOSTON CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2013
资助国家：
美国
起止时间：
2013-08-15 至 2016-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8866459
关键词：
5 year old Acute Pain Admission activity Adoption Adult Africa Age Applications Grants Arabs Area Benign Biological Biology Birth Boston Caring Cell Count Cell Therapy Chemicals Child Childhood Clinical Clinical Research Developed Countries Developing Countries Development Diagnosis Discipline Disease Environment Feasibility Studies Fetal Hemoglobin Foundations Genetic Globin Goals Haplotypes Health Expenditures Health Services Healthcare Fees Hematology Hemoglobin F Disease Hemoglobinopathies Histone Deacetylase Inhibitor Hospitals Human Immunization Individual Infant Mortality Institutes Institution Life Expectancy Mendelian disorder Metabolism Methods Minority Modality Molecular Morbidity - disease rate Nature Oral Organ Patients Pediatric Hospitals Penicillins Pharmaceutical Preparations Phenotype Pilot Projects Prophylactic treatment Public Health Research Research Personnel Sickle Cell Anemia Supportive care Symptoms Technology Toxic effect Translational Research Treatment Cost United Nations United States Woman World Health Organization base burden of illness chronic pain clinical investigation cost fetal gamma Globin gene therapy genetic approach improved innovation migration mortality multidisciplinary novel therapeutics nutrition population based pre-clinical programs research clinical testing sickling skills translational study

项目摘要

DESCRIPTION (provided by applicant): Sickle cell disease (SCD) is the most common hemoglobinopathy and one of the most common monogenic diseases in the world. SCD poses a major public health burden in the US with an aggregate charge for health care services of $1 billion. Globally, mortality, particularly in young children is very high. The goal of the proposed collaborative project is to develop new methods to treat SCD disease, with the realization that near term, high tech approaches involving genetic therapies applicable in the US will need to be replaced with inexpensive low-tech approaches utilizing targeted oral drugs that can be also be utilized in the developing areas of the world. Th focus of this application is thus to leverage state-of-the-art chemical and biological approaches to increase the expression of fetal hemoglobin to ameliorate the morbidity and cost of treatment of SCD. The proposed program seeks to take advantage of recent scientific breakthroughs, rapidly advancing technology and the environment of several outstanding research institutions to drive the translational agenda in this disease. The overarching theme of this multidisciplinary grant application is to utilize innovative scientific approaches to identify and put into clinical investigation new therapeutic modalities that modulate gamma-globin expression. There are three projects: i) integrating more traditional molecular hematology approaches, translational research, and approaches from the field of metabolism to identify new inducers of y-globin; ii) modulation of Bell 1A expression to increase HbF; iii) identifying HDAC inhibitors with optimal selectivity to maximize induction of y-globin expression while minimizing toxicity. Utilizing the established foundation of a highly sophisticated translational research program that focuses on clinical gene and cell therapy and anchors the Translational Research Skills Development Core, advice from world-renown senior investigators in SCD and globin biology at Children's Hospital Boston (CHB) and harnessing new scientific expertise not previously focusing on hemoglobinopathies at CHB, Brigham and Women's Hospital and the Broad Institute rapid migration of the scientific discoveries emanating from this program into pre-clinical and then clinical testing is likely.

描述（由申请人提供）：