Hematopoietic stem cells are an ideal vector for gene therapy since they repopulate the whole body, and in some cases can differentiate into various tissues in the body. This patent teaches the use of adeno-associated virus vectors to transfect hematopoietic stem cells so that the hematopoietic stem cells can repopulate the host and deliver the protein product of the gene to where it is needed.
Specific genes for transfection mentioned in the patent include:lysosomal glucocerebrosidase, beta globin and alpha 1 antitrypsin.
View this patent on the USPTO website.
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