Bone Marrow Failure | CHOP Research Institute

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Could an In Vivo Gene Therapy Strategy be Effective in Treating Fanconi Anemia?

Published on

Feb 24, 2025

Researching Gene Therapy as a Treatment for Fanconi Anemia

A pre-clinical study sets the stage for medical advances: A gene therapy treatment approach for Fanconi anemia patients.

Julia T. Warren, MD, PhD

Dr. Warren studies normal and aberrant hematopoiesis, with a special interest in granulopoiesis. Her research aims to understand the pathogenesis of severe congenital neutropenia and its risk for malignant transformation. She has a clinical interest in neutropenia syndromes including clinical trials.

E-mail:

warrenjt [at] chop.edu

Olson Laboratory

The Olson Lab aims to enhance diagnostics and treatment for bone marrow failure syndromes and improve outcomes of hematopoietic stem cell transplantation.

Bone Marrow Failure Research Study and Repository

To identify the underlying causes of bone marrow failure diseases for the purpose of developing and modifying treatment and diagnosis procedures.

Comprehensive Bone Marrow Failure Center

The Comprehensive Bone Marrow Failure Center brings together physicians and researchers to provide state-of-the-art diagnostics, clinical care, and investigational studies, as well as basic research focused on acquired and inherited bone marrow failure.

Timothy S. Olson, MD, PhD

Dr. Olson aims to improve diagnostics and treatment of bone marrow failure (BMF) syndromes, and to improve clinical hematopoietic stem cell transplantation (HSCT) outcomes. He conducts clinical trials of HSCT for non-malignant hematologic diseases. His laboratory explores both basic and translational research focused on genomics of BMF and the impact of BMF on hematopoietic niche function during HSCT.

E-mail:

olsont [at] chop.edu