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Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. The results were published in Haematologica.

/PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell...

Date Time Less is More for Next Generation of CAR T Cells PHILADELPHIA—When researchers from Penn Medicine found that many patients with B-cell acute lymphoblastic leukemia (ALL) treated with the investigational chimeric antigen receptor (CAR) T cell therapy targeting the CD22 antigen didn’t respond, they went back to the drawing board to determine why. They discovered that less is more when it comes to the length of what is known as the single-chain variable fragment — the linker that bridges the two halves of the receptor that allows CAR T cells to latch onto tumor cells and attack th...

Date Time 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 s...

 E-Mail 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 The...