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MOUNT SINAI PILOT CENTER FOR PRECISION DISEASE MODELING

Director: Ross L. Cagan, PhD One of three NIH-funded Pilot Centers (NIH-1U54OD020351) 

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Description.

OVERVIEW OF OUR PLATFORM

Genetics-based diseases have proven difficult to treat due to the complexity of the disease in the context of the whole body. At the Mount Sinai Pilot Center for Precision Disease Modeling, we are working to provide to the community a novel discovery platform designed to develop drugs and identify truly personalized therapeutics in a rapid, rational, and cost effective manner.

The Center leverages the advantages of the fruit fly Drosophila to build a ‘functional network platform’ designed to identify therapeutics that address disease complexity. We then use stem cell approaches to explore the most promising leads in a human cell context. Our overall objective is to further develop a platform and pipeline that can be adapted to a broad range of diseases.

We are currently focused on two diseases—colorectal cancer and RASopathy—to develop and demonstrate the strength of our platform to address both a rare Mendelian disease and one of the most common cancers. As our platform matures, we will offer a readily accessible standard operating procedure that will most efficiently bring our platform to the applied science community.

PROJECT LEADERS

Ross Cagan, PhD

Project 1

Advancing Drosophila Platforms for Therapeutics Discovery.

Arvin Dar, PhD

Project 2

Novel Approach to Developing Polypharmacological Lead Compounds.

Bruce Gelb, MD

Project 3

Using Human Induced Pluripotent Stem Cells for Drug Discovery in the RASopathies.

Carlos Cordon-Cardo, MD, PhD

Project 4

PDX-TIC: A Novel Platform for Discovery and Validation of Therapeutic Targets.

STEERING COMMITTEE MEMBERS

  • Marshall Posner, MD
    Professor of Medicine, Medical Director of the Head and Neck Oncology Center, and Associate Director of the Center for Personalized Cancer Therapeutics at the Mount Sinai Medical Center and the Icahn School of Medicine at Mount Sinai.
  • Robert Devita, PhD
    Professor in the Departments of Pharmacology and Systems Therapeutics and Structural and Chemical Biology and the Director of the Medicinal Chemistry Core of the Experimental Therapeutics Institute at the the Icahn School of Medicine at Mount Sinai
  • Marek Mlodzik, PhD
    Professor and Chair, Developmental and Regenerative Biology at the the Icahn School of Medicine at Mount Sinai


EXTERNAL ADVISORY BOARD MEMBERS

  • Nancy Bonini, PhD
    Florence R.C. Murray Professor of Biology at the University of Pennsylvania
  • Frank McCormick, PhD, FRS, DSc
    Professor Emeritus, UCSF Helen Diller Family Comprehensive Cancer Center, David A. Wood Distinguished Professorship of Tumor Biology and Cancer Research
  • Benjamin Neel, MD, PhD
    Professor, Department of Medicine and Director of the Laura and Isaac Perlmutter Cancer Center
  • Norbert Perrimon, PhD
    Professor of Genetics at Harvard Medical School and Investigator, Howard Hughes Medical Institute
  • Owen Sansom, PhD
    Professor and Deputy Director, Beatson Institute for Cancer Research

ADDRESS
1468 Madison Avenue
Annenberg Bldg. 25-40
New York, NY 10029

CONTACT
Email: ross.cagan@mssm.edu
Phone: 212-241-1427
Fax: 212-860-9279