# Gene Delivery Core

> **NIH NIH U54** · CINCINNATI CHILDRENS HOSP MED CTR · 2021 · $97,526

## Abstract

Abstract
Investigation of the molecular and cellular pathophysiology for hematologic diseases requires rapid and
low-cost access to vector preparations, and access to large libraries of vectors and production of vectors
destined to high throughput screening. Further, the successful development and testing of novel gene
therapies requires access to a translational vector “pipeline” which necessitates a close interaction
between basic research, research vector and clinical vector manufacturing laboratories. Cincinnati
Children’s Hospital Medical Center, a clinical and research institution of excellence, has developed
outstanding cores to provide hematology investigators with vector support for biological studies for the
genetic manipulation of cells and their translation to the clinic. This core has a track record of providing
high quality “research-grade” and “clinical-scale” vector services to investigators at various stages of basic
and translational research, ensuring expeditious translation to the clinic. The components of the Gene
Delivery Core (GDC) that contribute to this effort are the Viral Vector Core and Vector Library Core. Each
of these sub-cores are equipped with its own state-of-the-art research laboratories to allow rapid access
to vector libraries and viral vector products, their evaluation in routine basic research lab studies and/or
high throughput screens, and their translation to the clinic as the studies move through the translational
“pipeline”. All of this effort is backed by a strong Institutional commitment to translational research and
widely popular demands from users in the broad hematology field. Our objectives are to provide: (1)
research scale and medium scale pilot and feasibility viral vector products and consultation on vector type
and design, and (2) vector library core services including production of lentiviral shRNA and CRISPR
gRNA libraries for high throughput screening. Our overall goal is to advance research and development in
hematology via facilitating gene delivery, gene knock down, gene knockout or gene editing, to accelerate
the rate of translation.

## Key facts

- **NIH application ID:** 10201889
- **Project number:** 1U54DK126108-01A1
- **Recipient organization:** CINCINNATI CHILDRENS HOSP MED CTR
- **Principal Investigator:** Jose A. Cancelas
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $97,526
- **Award type:** 1
- **Project period:** 2021-08-01 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10201889

## Citation

> US National Institutes of Health, RePORTER application 10201889, Gene Delivery Core (1U54DK126108-01A1). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10201889. Licensed CC0.

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