Researchers at Children's Hospital of Philadelphia Find Lentiviral Gene Transfer Improves Human Alpha Globin Production for the Treatment of Alpha Thalassemia
Innovative model highlights potential platform to develop novel therapies
Thousands of children are born with AT every year, especially in
"New treatments for blood disorders have experienced remarkable success in recent years, particularly for conditions like beta thalassemia and sickle cell disease. However, despite representing a growing healthcare challenge, Alpha Thalassemia has drawn significantly less attention," said senior author
Allogeneic bone marrow transplantation (BMT) is currently the only available therapeutic option for patients with severe AT, and that method also requires an appropriate donor. Additionally, until now, research into therapeutic advancements for AT was limited due in part to the challenge of creating adult mouse models with this disease.
In this study, researchers designed an innovative model by deleting alpha globin genes in adult mice using a lipid nanoparticle (LNP) embedded within mRNA that induced the deletion of the alpha globin genes. This led to the production of faulty red blood cells (RBC) with abnormal hemoglobin, called HbH, that binds oxygen tightly, preventing its delivery to tissues and causing paradoxical hypoxia. The targeted LNP platform technology was previously established by Hamideh Parhiz, PharmD, PhD, a co-senior study author and Assistant Professor of Medicine at Penn Medicine. Her team also generated targeted LNP to hematopoietic stem cells encapsulating mRNA for the current study.
Once the alpha globin genes were deleted, the mice experienced decreased oxygen levels comparable to individuals with severe AT. The mice were flooded with RBC that couldn't transport oxygen, posing an extreme health threat. The researchers confirmed that this model can now be applied to test novel or genetic therapies in human patients to improve their clinical care.
Rivella, along with members of his lab, including,
"This innovative approach represents a much-needed step forward in the treatment of Alpha Thalassemia," said Rivella. "We look forward to further research and the promise of improved patient outcomes with fewer complications over time."
The authors would like to acknowledge the Pathology Core Laboratory at CHOP's Research Institute for providing histological, immunohistochemical, and imaging services. The research was funded by
Rivella, et. al. "Use of HSC Targeted LNP to Generate a Mouse Model of Lethal α-Thalassemia and Treatment via Lentiviral Gene Therapy." Blood. Online
About Children's Hospital of Philadelphia:
A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout
Contact:
Children's Hospital of Philadelphia
(267) 426-6084
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SOURCE Children's Hospital of Philadelphia
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