Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on alpha-Thalassemia

MeSH tree: C16.320.070.875.100

One-time gene therapy could end lifelong transfusions for rare blood disease

Thanks to in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a growing population of children born ...

Sep 18, 2025

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Lentiviral gene transfer improves human alpha globin production for the treatment of alpha thalassemia

Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine have pioneered a new model that offers a potential platform for developing novel therapies to treat ...

Jul 2, 2024

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Uncovering thalassemia diversity in southern China through next-generation sequencing

Around 5.2% of the global population carries abnormal hemoglobin genes. Each year, 300,000 to 500,000 children are born with severe hemoglobinopathies worldwide, with approximately 80% of these cases occurring in developing ...

Sep 4, 2023

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