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.
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.