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Program Snapshot

The goal of the Regenerative Medicine Program (RMP) is to serve as a national resource for stem cell science to accelerate the development of new medical applications and cell-based therapies. The RMP is pursuing this goal through two major initiatives; a Therapeutic Challenge Award to Dr. Kapil Bharti at the National Eye Institute (NEI) and a recently established Stem Cell Translation Laboratory (SCTL) within the National Center for Advancing Translational Sciences (NCATS).

Dr. Bharti’s research focuses on developing a stem-cell based therapy to treat age-related macular degeneration, a leading cause of blindness in the elderly; and the methodological and regulatory challenges that must be overcome to move his research beyond the pilot stage toward the clinic is relevant to the scientific and clinical communities. The SCTL is designed to tackle top scientific and technological problems that currently impede therapeutic use of induced pluripotent stem cells (iPSCs) — adult cells reprogrammed to behave similarly to embryonic stem cells — and to rapidly deliver the resulting protocols, standards, data, tools and molecules to the broader scientific community.

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Program Highlights

Two-year update on RMP Therapeutic Challenge Award to Dr. Kapil Bharti
On September 18, 2015 Dr. Kapil Bharti of the National Eye Institute and his research team presented an update to his Therapeutic Challenge award to develop a stem-cell based therapy to treat age-related macular degeneration (AMD). In AMD patients Retinal Pigment Epithelium (RPE) cells become damaged and lose function. Replacement of damaged RPE cells with functional stem-cell-derived RPE cells is a viable treatment believed to restore or improve vision. Inducible pluripotent stem cells (iPSCs) are stem cells that have been reprogramed from adult cells and can develop into any cell type in the body. For clinical applications iPSCs are to be manufactured using strict guidelines called current good manufacturing practice (cGMP) to ensure quality, purity, and safety of cells. Dr. Bharti’s team has developed a streamlined manufacturing process to generate transplant-ready cGMP-grade RPE cells from iPSCs and the entire manufacturing process takes slightly under 150 days. His team is currently performing pre-clinical animal toxicity, efficacy, and transplantation device compatibility studies in preparation for submitting a phase I Investigational New Drug (IND) application with the Food and Drug Administration in 2017. Learn more about Dr. Bharti’s research.

Research articles from Dr. Bharti’s Therapeutic Challenge Award

  1. Hotaling, N.A., Bharti, K., Kriel, H., and Simon, C.G. (2015) Validated open source nanofiber diameter measurement tool. Biomaterials. 61: 327-338.
  2. Hotaling, N.A., Bharti, K., Kriel, H., and Simon, C.G. (2015) Dataset for the validation and use of DiameterJ an open source nanofiber diameter measurement tool. Data In Brief. 5: 13-22.
View the National Centers for Biomedical Computing (NCBC) Showcase!

Learn more about the research, training, and outreach in computational science offered through the Bioinformatics and Computational Biology program.

View the NCBC Showcase Day 1, and Day 2.

 

National Centers for Biomedical Computing

 

Select an icon below to learn more about the history, research impact, resources, and training for each collaboration.

 

National Center for Simulation of Biological Structures (SimBioS) at Stanford University
National Center for Simulation of
Biological Structures
at Stanford University
Center Summary

National Center for the Multiscale Analysis of Genomic and Cellular Networks (MAGNet) at Columbia University
National Center for Multiscale Analysis
of Genomic and Cellular Networks
(MAGNet) at Columbia University
Center Summary
National Alliance for Medical Image Computing (NA-MIC) at Brigham and Women's Hospital
National Center for Medical Image
Computing (NA-MIC) at Brigham and
Women's Hospital
Center Summary
Integrating Biology and the Bedside (I2B2) at Brigham and Women's Hospital
Integrating Biology and the Bedside (I2B2)
at Brigham and Women's Hospital
Center Summary
National Center for Biomedical Ontology (NCBO) at Stanford University
National Center for Biomedical Ontology
(NCBO) at Stanford University
Center Summary
Integrate Data for Analysis, Anonymization, and Sharing (IDASH) at the University of California, San Diego
Integrate Data for Analysis,
Anonymization and Sharing (IDASH) at
the University of California, San Diego
Center Summary

 

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