# Mouse Gene Manipulation Core

> **NIH NIH U54** · BOSTON CHILDREN'S HOSPITAL · 2020 · $167,869

## Abstract

MOUSE GENE MANIPULATION CORE (CORE E) 
ABSTRACT 
The primary objective of the Mouse Gene Manipulation Core is to provide all our IDDRC 
investigators with a centralized, affordable and quality-controlled service, using state-of-the art 
genome editing technology, for the rapid and efficient generation of genetically altered mouse 
lines. Our goal is aid our PIs in their work on identifying the role of particular genes in the 
development of the nervous system and in modelling intellectual disability and 
neurodevelopmental disorders, with a view in particular of identifying preclinically, novel 
biomarkers of these disorders and for evaluating the efficacy of new therapeutic approaches. 
Although manipulation of the mouse genome has been well established for several decades 
using homologous combination in embryonic stem cells for gene targeting and random insertion 
of DNA in zygotes for making transgenic mice, and this Core has very effectively used these 
approaches to generate many mouse models of neurodevelopmental disorders, during the 
course of the present grant cycle transformative advances have been made in our capacity for 
genome editing. This core has enthusiastically embraced and mastered the new CRISPR/Cas9 
technology for disrupting gene expression by base insertion/deletion (INDELS), and using 
homology directed repair (HDR) for introducing mutations, inserting reporters, Cre or Flp drivers 
and indeed making many kinds of changes to the genome – in a manner that is both efficient 
and fast. We will continue to offer our standard technology since there are users and uses that 
favor this but anticipate that CRISPR/Cas9 and its evolution will largely take over. The 
technology has caused immense excitement in the IDDRC community and the demand for our 
services are set to increase substantially, so that the IDDRC can provide parallel preclinical and 
clinical phenotyping and efficacy studies. To aid in the uptake of genome editing the core will 
offer consultation services on the best approach for PIs to use for particular projects, advice on 
selection of guide RNAs, genotyping and colony management, as well as a new 
cryopreservation service. Collectively the new services will offer the IDDRC mouse models 
faster and cheaper with the capacity to preserve these for alter use or sharing.

## Key facts

- **NIH application ID:** 10003045
- **Project number:** 5U54HD090255-05
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** CLIFFORD J WOOLF
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $167,869
- **Award type:** 5
- **Project period:** — → —

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10003045

## Citation

> US National Institutes of Health, RePORTER application 10003045, Mouse Gene Manipulation Core (5U54HD090255-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10003045. Licensed CC0.

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