Transgenic non-human mammals as models for human pathologies of stem cell origin

Patent Number: 7,456,333

Chronic myeloid leukemia is characterized by a chromosomal translocation resulting in production of the bcr-abl fusion protein, which is believed to be responsible for initiation of the malignancy.  Vaccination against bcr-abl peptides has been associated with some clinical benefit, and small molecule targeting of this protein has resulted in successful inductions of remission in patients.

The current patent covers a mouse model of CML in which the bcr-abl protein product is expressed transgenically only in cells possessing the stem cell antigen (SCA-1), which is associated with hematopoietic stem cells. 

The mouse develops a CML-like disease that is characterized by a chronic and accelerated phase.

Interestingly SCA-1 is found on other cells besides hematopoietic stem cells, including renal stem cells and liver progenitors.  Therefore it will be interesting to see if this mouse has other non-hematological abnormalities. 

View this patent on the USPTO website.