A genetically engineered clotting factor that controlled hemophilia in an animal study offers a novel potential treatment for human hemophilia and a broad range of other bleeding problems.
Division of Hematology Research
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CHOP researcher uses adeno-associated virus (AAV) vectors in clinical trials of gene therapy for hemophilia B.
Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated hemophilia, a blood clotting disorder, in mice.
Hematopoietic stem cell transplantation in utero (IUHSCTx) is a promising approach for the treatment of a variety of hematologic disorders, including sickle cell disease, CHOP researchers say.