# A National iPS Cell Network with Deep Phenotyping for Translational Research

> **NIH NIH U01** · BOSTON UNIVERSITY MEDICAL CAMPUS · 2020 · $799,470

## Abstract

Project Summary/Abstract
The discovery of iPSCs provides an unprecedented opportunity for any scientist to derive an
inexhaustible supply of patient-derived primary cells. These cells containing each patient's own genetic
background can now be applied for in vitro human disease modeling, drug screening of personalized
therapeutics, and the development of future regenerative cell-based therapies. The most valuable human
clones already generated by the CTSA investigators collaborating on this proposal not only carry common
disease-associated mutations and polymorphisms, but also carry knock-in fluorochrome reporters
targeted to specific loci through state-of-the-art gene editing technologies. The goal of this proposal is
the establishment of a CTSA network of induced pluripotent stem cell (iPSC) repositories and
iPSC cores that will enable advanced disease modeling using >1000 existing normal and disease
specific human cell lines and banking 6,000 additional samples procured from the 2nd and 3rd
generation participants of the Framingham Study.
A concerted effort for curation, sharing, and distribution of this vital resource across all CTSAs does not
exist. This proposal thus creates a CTSA iPSC Network led by teams who have championed an `Open
Source Biology' approach, freely sharing iPSC lines and their reprogramming reagents with more than
500 labs to date across the globe. Its goals are to make patient-derived iPSCs together with the tools
and expertise for their genetic manipulation available to the greater research community on a large scale
to realize their promise for extending understanding of disease and developing potential therapies. To
achieve these goals, it proposes: a) national sharing of >1000 iPSC lines already derived by the CTSA
teams collaborating in this proposal, representing a critical resource in high demand by both basic and
clinical researchers, b) development and support of formalized education and training programs able to
nationally disseminate the expertise required to fully harness these new tools and differentiate them into
the wide diversity of human cell lineages, c) maintenance and sharing of open source gene-editing tools
and gene edited iPSC lines that will enable CTSA investigators to manipulate the human genome at will,
and d) derivation for national sharing of additional iPSC lines generated from the most densely clinically
and genetically phenotyped cohort of individuals currently followed in the USA today: the ~6,000
participants of the second and third generations of the Framingham Study.

## Key facts

- **NIH application ID:** 9996816
- **Project number:** 5U01TR001810-05
- **Recipient organization:** BOSTON UNIVERSITY MEDICAL CAMPUS
- **Principal Investigator:** Yoav Gilad
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $799,470
- **Award type:** 5
- **Project period:** 2016-09-15 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9996816

## Citation

> US National Institutes of Health, RePORTER application 9996816, A National iPS Cell Network with Deep Phenotyping for Translational Research (5U01TR001810-05). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/9996816. Licensed CC0.

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