Our Breakthrough

Engineering a Superior Gene Therapy Vector

A safer, more effective vector could offer a new treatment option for patients with devastating blood disorders

Sickle Cells

Our Challenge

A Disease Without a Cure

Blood disorders like sickle cell disease and beta-thalassemia wreak havoc on a child’s body, causing anemia and excruciating pain and increasing the risk of life-threatening infections. Treatment options are limited, and existing gene therapy can come with toxic side effects.

Our Solution

Building a Better Vector

Through donation-supported research, Stefano Rivella, PhD, and Laura Breda, PhD, have engineered a superior vector that does more with less. By requiring less vector to deliver healthy genes to a patient’s cells, this new technology reduces the potential of bone marrow defects and cancer. This stronger vector could also lead to modifications that reduce the side effects associated with the myeloablation required to engraft genetically corrected bone marrow cells. Further development of this technology could lead to in vivo gene delivery, thus eliminating the need for toxic myeloablation altogether.

Read about the study

CHOP Researchers
Dallas Sickle Cell Patient Smiling Behind a Mask

Our Impact

A Potentially Curative Therapy

This new vector, soon moving into clinical trials, could provide a safer, more effective treatment for patients with sickle cell disease.

Meet Dallas

Our Team

Dedicated to Changing Lives

Dr. Rivella and his team are leaders in blood disorder research and treatment.

Stefano Rivella, PhD bio photo

Stefano Rivella, PhD

Professor of Pediatrics, Division of Hematology & Scientific Director of the Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)

Laura Breda, PhD bio photo

Laura Breda, PhD

Research Assistant Professor, Division of Hematology

Moving Ahead

Paving a Path Forward

Upon FDA approval, this new vector could potentially offer a curative therapy for serious blood disorders that — for many children — have long lacked viable treatment options.

Help Us Find Solutions

We find real-world, scalable solutions to seemingly intractable problems. Led by the greatest minds in medicine, and with support from our generous donors, our world-renowned teams are taking on the toughest diseases the world has ever seen – and winning.

Scientific image collage