Hematopoietic Stem Cells | CHOP Research Institute

‘Medicine of the Future’: Delivering mRNA Gene Editing Tools In Vivo

Published on

Jul 31, 2023

‘Medicine of the Future’ in Development at CHOP to Treat Blood Disorders

Researchers developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

First Potentially Curative Gene Therapy for Beta Thalassemia: Q&A with Janet Kwiatkowski, MD, MSCE

Published on

Sep 9, 2022

in Cornerstone Blog

In this Q&A, hematologist Dr. Kwiatkowski discusses the Food and Drug Administration approval of beti-cel (Zynteglo®), a gene therapy for beta thalassemia.

Tong Laboratory

The Tong laboratory studies cytokine receptor and ubiquitin signaling in hematopoietic stem cells, bone marrow failure, and leukemia.

Rivella Laboratory

The Rivella Laboratory uses lentiviral vectors for modulation of gene expression and gene transfer for the cure of hemoglobinopathies.

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.

Kurre Laboratory

The Kurre Laboratory has a longstanding interest in Fanconi Anemia (FA), a genetic condition with prominent hematologic complications.

Wei Tong, PhD

Dr. Tong investigates cytokine receptor signaling in normal and neoplastic hematopoietic development. She uses integrated approaches encompassing biochemistry, molecular biology, mouse models, and primary human samples to understand signaling events emanating from cytokine receptors that regulate the development of hematopoietic stem/progenitor cells.

E-mail:

tongw [at] chop.edu

Peter Kurre, MD

Dr. Kurre's laboratory has longstanding expertise in Fanconi Anemia (FA), a genetic condition with prominent hematologic complications. With training in transplantation and hematopoietic stem cell biology, he hopes to improve the understanding of the progressive hematopoietic failure in patients with bone marrow failure and FA, broaden diagnostic approaches, and develop next generation therapies.

E-mail:

kurrep [at] chop.edu