Hemoglobinopathies | CHOP Research Institute
 

Hemoglobinopathies

Stem Cell Transplant for Sickle Cell Disease or Thalassemia Major

Stem Cell Transplant for Sickle Cell Disease or Thalassemia Major

The study will enroll male and female patients between 2 years to 25 years of age with sickle cell disease or beta thalassemia major, who do not have a matched related donor, and their doctor feels their condition should be treated with hematopoietic stem cell transplantation (HSCT).

Rivella Laboratory

Rivella Lab

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

Abdulmalik Laboratory

Abdulmalik Lab

Research in the Abdulmalik Laboratory focuses on the development of novel therapies for the hemoglobinopathies, with a particular emphasis on developing and validating novel therapeutic agents for sickle cell disease.

Will a New Vector Offer Safer Gene Therapy for Red Blood Disorders?

Published on
Feb 1, 2021
in Cornerstone Blog
Snapshot Science: Will a New Vector Offer Safer Gene Therapy for Red Blood Disorders?
CHOP researchers have developed a potentially safer and more effective gene therapy vector for red blood disorders.

Exploring Exosome-Mediated Stem Cell Engraftment for Hemoglobinopathies

Published on
May 8, 2019
in Cornerstone Blog
Meghana V. Kashyap, MD
A surgeon-scientist explores exosome-mediated stem cell engraftment as a means to lessen disease severity and enhance quality of life for children with hemoglobinopathies.
Jason L. Freedman, MD, MSCE
Jason L. Freedman

Dr. Freedman has a particular interest in epidemiologic and translational research within pediatric supportive oncology. He aims to advance the understanding and treatment of symptoms, infections, and organ toxicities in pediatric cancer and hematopoietic stem cell transplant patients.

E-mail:
freedmanj [at] chop.edu
Stefano Rivella, PhD
Stefano Rivella

Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies. He also investigates additional disorders such as anemia of inflammation and hemochromatosis.

E-mail:
rivellas [at] chop.edu