Project Details
Description
The General Research Center (GCRC) serves as the physical and
intellectual focus for clinical research endeavors at the Mount Sinai
School of Medicine (MSSM). For the past 32 years, the GCRC has provided
the essential resource to: 1) elucidate the pathophysiologic mechanisms
of a variety of diseases and 2) to evaluate laboratory advances at the
bedside by determining the safety and efficacy of new or improved
modalities for their diagnosis, treatment and/or prevention. In addition,
the GCRC is the primary training site for clinical investigators in the
Physician/Scientist tradition. Continuation of this invaluable research
and training resource will permit the accomplishment of on-going studies
by investigators from a wide variety of clinical disciplines, e.g.,
Community Medicine, (Geriatrics, Human Genetics, Medicine, Neoplastics,
Neurology, Ob/Gyn, Ophthalmology, Pediatrics, Psychiatry, and Surgery.
Efforts are continously underway to further enhance the use of the GCRC
research resources by recruiting new Investigators and high quality
research protocols. Emphasis will be directed to recruit and coordinate
protocols in three areas: 1) molecular medicine and the development of
molecular-based therapies, especially cellular and gene therapies 2)
research in AIDS directed to pathogenesis, treatment and prevention and
3) biologic psychiatry with emphasis on shizophrenia and Alzheimer's
Disease. Examples of such research studies include: 1) Development
and/or evaluation of gene-product replacement therapies for lysosomal
storage disease using recombinant enzyme replacement therapy in Fabry,
Gaucher Type 1, and Niemann-Pick B diseases, 2) Elucidation of the
molecular basis of phenotypic variation and the identification of
genotype/phenotype correlations for several inherited disorders, 3)
Molecular studies of the ALA-dehydratase polymorphism and genetic
susceptibility to lead toxicity, 4) Development and evaluation of
pharmacologic and gene therapy strategies to the treatment of genetic
disorders, particularly sickle cell disease, Niemann-Pick B disease, and
Glanzmann's thrombasthenia, 5) Studies of the etiology and treatment of
Alzheimer's disease and psychiatric disorders, 6) Evaluation of cellular
therapy in Parkinson's disease by fetal nigral cell transplantation, and
7) Evaluation of various approaches for the treatment and prevention of
AIDS. To facilitate these studies, the Core Laboratory will continue to
provide molecular biology expertise and consultation and offer GCRC
investigators a variety of services such as establishment of fibroblast
and lymphoblast lines, DNA extraction, Southern and northern analyses and
PCR-based assays for common mutations or polymorphisms, including the use
of robotics to automate repetitive assays. In addition, the Core
Laboratory will increase its capabilities as the Gene Therapy Assessment
Laboratory in order to monitor cell and gene therapy endeavors by semi-
quantative RT-PCR, in situ hybridization, and other gene and protein
detection techniques. The proposed new directions and laboratory
capabilities should provide the clinical research environment for the
anticipated medical advances resulting from molecular revolution.
Status | Finished |
---|---|
Effective start/end date | 12/1/99 → 2/28/01 |
ASJC
- Applied Microbiology and Biotechnology
- Clinical Neurology
- Genetics(clinical)
- Neurology
- Medical Laboratory Technology
- Infectious Diseases
- Genetics
- Molecular Medicine
- Obstetrics and Gynecology
- Maternity and Midwifery
- Biotechnology
- Toxicology
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