Transplantation of Endothelial Cells
Description: PUBLIC HEALTH RELEVANCE: Liver-directed cell therapy has extensive potential for impacting human health. Already, some 100 people have undergone hepatocyte transplantation for liver failure, enzyme deficiency states, FVII deficiency, etc. (1-3). Moreover, animal studies showed that cell therapy will be helpful in other situations. Based on our work under this grant in transplantation of endothelial cells, extensive further interest has been generated in cell therapy for coagulation defects, e.g., hemophilia. Our cell transplantation studies in hemophilia mouse have shown for the first time that hemophilia can be permanently cured by cell therapy. Despite work over the past two decades, this accomplishment in hemophilia had eluded gene therapy investigators. Since hemophilia A afflicts over one million individuals worldwide, this will be highly significant for human health. Moreover, many individuals with hemophilia are burdened with associated liver diseases, e.g., chronic hepatitis B and C. Therefore, replacement of healthy LSEC and hepatocytes will eventually be appropriate for many individuals. In this way, lessons from the current proposal will be of extensive value for larger populations, since chronic viral hepatitis afflicts some 400 million people worldwide, and will have uncommon public health significance and impact in therapies for liver diseases.
Funding type: research
Funder scheme: R01
Start date: 2006-04-01
End date: 2019-04-30
Investigator: SANJEEV GUPTA
Institution: ALBERT EINSTEIN COLLEGE OF MEDICINE
Funded outputs count: 60
Funder award ID: R01DK071111
OpenAlex ID: G7102017808