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CHOP Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders New preclinical study shows vector results in significantly more hemoglobin production than vectors currently used in gene therapy for sickle cell disease and beta-thalassemia News provided by Share this article Share this article PHILADELPHIA, Jan. 29, 2021 /PRNewswire/  Researchers at Children s Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that 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.

United statesKwame ohene frempongRivellas lentiviralStefano rivellaLaura bredaAssociazione venetaDepartment of health pennsylvaniaCommonwealth universal research enhancement programFrontier programKwame ohene frempong chairSickle cell anemiaComprehensive centerSickle cell diseaseRed cell disordersDana bateஒன்றுபட்டது மாநிலங்களில்