Researchers at the Children’s Hospital of Philadelphia are Improving Gene Therapy Vectors

The Children’s Hospital of Philadelphia: Improving Gene Therapy Vectors

Improving gene therapy vectors seems to have come at the hands of researchers at Children’s Hospital of Philadelphia (CHOP). A gene therapy vector was developed for blood disorders like sickle cell disease (SCD) and beta-thalassemia, stating that this vector is potentially safer and more effective than those currently used in gene therapy trials for those conditions.

The vector – an engineered vehicle for delivering functional copies of the hemoglobin gene to correct a genetic abnormality, leads to the production of more hemoglobin with a lower dose, minimizing the risk of toxic side effects.

The findings were published in Molecular Therapy.

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