Gene Delivery Core

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.