# Preclinical Models Core

> **NIH NIH P50** · CHILDREN'S HOSP OF PHILADELPHIA · 2021 · $189,108

## Abstract

(CORE F – PMC: PRECLINICAL MODELS CORE)
PROJECT SUMMARY
Description: The Preclinical Models Core (PMC) supports IDDRC users who study IDD pathophysiology and/or
seek new treatments for IDD using one or both of the following preclinical models: (1) Rodent models: This Core
component assists users with the characterization of behavioral phenotypes in mouse models of genetic or
acquired disabilities. Major behavioral domains assessed in this core include: learning, memory, social
preference, communication, motor function, and affective disorder-related behaviors; (2) Human iPSC models:
This Core component assists users to create cell models of IDD by generating induced pluripotent stem cells
(iPSCs) using standard reprogramming technologies or by genome editing via CRISPR-Cas9 on established
iPSC lines, followed by differentiation into neural cell types of interest for molecular and cellular phenotype
characterization. These cells, some derived from humans with IDDs, provide a model in which it is possible to
scrutinize molecular and cellular consequences of genetic disease and to evaluate the efficacy of possible
therapies. The complementary model systems are being used by many of the center investigators, and the two
services interact closely under one leadership.
The PMC emphasizes training of users and their staff. Trained users can continue studies in their own laboratory,
or they can utilize the equipment and/or reagents in the PMC at reduced cost. The services offered by the PMC
are complementary to those offered by all of the other center cores, and users frequently use more than one
core to complete a project. In addition, the PMC interacts closely with similar cores at sister IDDRCs to share
protocols, cross-validate outcome measures, in order to improve rigor and reproducibility across the IDDRC
network.
Relevance to IDDRC Mission: The PMC bridges all three domains of “Genes, Brain, and Behavior”, the theme
of the CHOP/Penn IDDRC (see Research Plan Overall). The Core was developed in response to a user survey
in 2014 that emphasized a need for an IDD-focused facility to study IDD-related behavioral and cellular
phenotypes in the context of IDD clinical symptoms and to take advantage of the iPSC potentials to gain insights
into disease pathophysiology and improve treatment for IDDs in a collaborative manner with other Cores of this
IDDRC.
Eligibility: These services are available both to approved users of the IDDRC at CHOP/Penn and to users at
other Centers in the Network.

## Key facts

- **NIH application ID:** 10240004
- **Project number:** 1P50HD105354-01
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** Zhaolan Zhou
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $189,108
- **Award type:** 1
- **Project period:** 2021-07-15 → 2026-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10240004

## Citation

> US National Institutes of Health, RePORTER application 10240004, Preclinical Models Core (1P50HD105354-01). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10240004. Licensed CC0.

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