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Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders

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Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders
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.
The findings were published today in Molecular Therapy.
“These results have many potential benefits for the successful treatment of patients affected by beta-globinopathies like sickle cell disease and beta-thalassemia, including a better dose response, a minimized chance of clonal expansion and tumorigenesis, a reduced cost of therapy, and a potentially reduced ....

United States , Kwame Ohene Frempong , Rivellas Lentiviral , Stefano Rivella , Laura Breda , Associazione Veneta , Department Of Health Pennsylvania , Commonwealth Universal Research Enhancement Program , Frontier Program , Kwame Ohene Frempong Chair , Sickle Cell Anemia , Comprehensive Center , Sickle Cell Disease , Red Cell Disorders , ஒன்றுபட்டது மாநிலங்களில் , லாரா ப்ரேடா , துறை ஆஃப் ஆரோக்கியம் பென்சில்வேனியா , காமன்வெல்த் உலகளாவிய ஆராய்ச்சி விரிவாக்கம் ப்ரோக்ர்யாம் , எல்லை ப்ரோக்ர்யாம் , அரிவாள் செல் இரத்த சோகை , விரிவான மையம் , அரிவாள் செல் நோய் , சிவப்பு செல் கோளாறுகள் ,

A potentially safer, more effective gene therapy vector for blood disorders

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Philadelphia, January 29, 2021 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.
The findings were published today in
Molecular Therapy.
These results have many potential benefits for the successful treatment of patients affected by beta-globinopathies like sickle cell disease and beta-thalassemia, including a better dose response, a minimized chance of clonal expansion and tumorigenesis, a reduced cost of therapy, and a potentially reduced need for chemotherapy or radiation before beginning gene therapy, ....

United States , Rivellas Lentiviral , Kwame Ohene Frempong , Stefano Rivella , Laura Breda , Associazione Veneta , Department Of Health Pennsylvania , Commonwealth Universal Research Enhancement Program , Frontier Program , Kwame Ohene Frempong Chair , Sickle Cell Anemia , Comprehensive Center , Sickle Cell Disease , Red Cell Disorders , ஒன்றுபட்டது மாநிலங்களில் , லாரா ப்ரேடா , துறை ஆஃப் ஆரோக்கியம் பென்சில்வேனியா , காமன்வெல்த் உலகளாவிய ஆராய்ச்சி விரிவாக்கம் ப்ரோக்ர்யாம் , எல்லை ப்ரோக்ர்யாம் , அரிவாள் செல் இரத்த சோகை , விரிவான மையம் , அரிவாள் செல் நோய் , சிவப்பு செல் கோளாறுகள் ,

CHOP Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders

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
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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 States , Kwame Ohene Frempong , Rivellas Lentiviral , Stefano Rivella , Laura Breda , Associazione Veneta , Department Of Health Pennsylvania , Commonwealth Universal Research Enhancement Program , Frontier Program , Kwame Ohene Frempong Chair , Sickle Cell Anemia , Comprehensive Center , Sickle Cell Disease , Red Cell Disorders , Dana Bate , ஒன்றுபட்டது மாநிலங்களில் , லாரா ப்ரேடா , துறை ஆஃப் ஆரோக்கியம் பென்சில்வேனியா , காமன்வெல்த் உலகளாவிய ஆராய்ச்சி விரிவாக்கம் ப்ரோக்ர்யாம் , எல்லை ப்ரோக்ர்யாம் , அரிவாள் செல் இரத்த சோகை , விரிவான மையம் , அரிவாள் செல் நோய் , சிவப்பு செல் கோளாறுகள் , டானா பேட் ,