Description:
Salk Test
INVENTION: Salk Institute scientists identified highly efficient methods and compositions for making and using disease-free patient specific iPS cells. These iPS cells are generated through the correction of genetic defect(s) in somatic cells, such as keratinocytes, from patients with bone marrow failure diseases, reprogrammed to pluripotency without c-MYC, and give rise to hematopoietic progenitors of peripheral blood cells that are disease free. This iPS cell technology could also be used for generation of patient-specific disease-corrected cells for treatment of diseases involving decline in stem cells numbers.
APPLICATIONS:
• Immunodeficiency diseases, thalassaemia, sickle-cell anemia, Fanconi anaemia, haemophilia A, haemophilia B, cycstic fibrosis, a1-antitrypsin deficiency, Canavan disease, muscular dystrophy, adenosine deaminase deficiency, Tay Sachs disease, Fragile X chromosome, Hungton’s disease, Gaucher’s disease, Hurler’s disease, von Recklinghausen’s disease, familial hypercholesterolemia, von Willebrand disease, Congenital leptin deficiency, Congenital neurologic diabetes insipidus, Fabry disease, and Pompe disease
• Experimental platform to model various human disease
ADVANTAGES:
• C-MYC not required for reprogramming
• Safer and more efficacious than genetic correction of autologous hematopoietic stem cells (HSC) with integrative vectors
• Results in production of large numbers of autologous HSCs used to restore hematopoietic function
• Only long term effective treatment for Fanconi Anaemia
STAGE OF DEVELOPMENT: Early stage, preclinical animal data for Fanconi Anaemia
BACKGROUND: The possibility of reprogramming mature somatic cells to generate induced pluripotent stem (iPS) cells may have a wide range of applications in cell and gene therapy, and could be particularly relevant for the treatment of diseases involving decline in hematopoietic stem cell numbers. Generation of disease-free hematopoietic progenitor cells from genetically corrected reprogrammed cells from other tissues opens a new therapeutic option for such diseases.
INVENTORS: Juan Carlos Izpisua Belmonte, Inder Verma, Juan Bueren, and Angela Raya
PATENT STATUS: pending applications: US (12/789,375), Australia, Canada, China, Europe, Israel, India, Japan, South Korea, Russia, Singapore
CONTACT: Michelle A. Booden, Ph.D., 858.453.4100 x1612, mbooden@salk.edu
REFERENCE #: S09017