DESCRIPTION (provided by applicant): Hemophilia A is a bleeding disorder caused by the absence of clotting factor VIII (FVIII). This disease is inherited in an X-linked recessive manner and it is estimated that there are 15,000 affected individuals in the U.S. Treatment of Hemophilia A has been… More

DESCRIPTION (provided by applicant): Fanconi anemia (FA) is an inherited recessive disorder caused by deficiency of one of several (up to 9) proteins involved in the regulation of DNA repair. Patients exhibit birth defects, suffer from bone marrow failure early in life, and are prone to develop… More

DESCRIPTION (provided by applicant): The American Cancer Society predicts that over 1.3 million new cases of cancer will be diagnosed in 2003. With a 5-year relative survival rate at only 62% for all cancers combined, new therapies are clearly needed. Critical to tumor growth and metastasis is… More

DESCRIPTION (provided by applicant): Hemophilia A is a bleeding disorder caused by the absence of clotting factor VIII (FVIII). This disease is inherited in an X-linked recessive manner. There are 15,000 affected individuals in the U.S. Treatment of Hemophilia A is based on frequent delivery of… More

DESCRIPTION (provided by applicant): Discovery Genomics, Inc. is focused on development of the Sleeping Beauty (SB) transposon system as a non-viral means of integrating new gene sequences in cells and tissues for therapeutic purposes. Here we propose to d evelop a method for improved targeting of… More

DESCRIPTION (provided by applicant): Fanconi anemia (FA) is a rare, autosomal recessive inherited disease caused by the absence of any of eight different proteins that regulate DNA repair. Affected individuals suffer from bone marrow failure early in life. FA can be effectively treated by… More

DESCRIPTION (provided by applicant): The overall goal of Discovery Genomics, Inc. is to develop the Sleeping Beauty transposon system for human gene therapy. To achieve this to treat hemophilia, we must be able to direct uptake and long-term expression of therapeutic genes in targeted tissues such… More

DESCRIPTION (provided by applicant): Primary immune deficiencies comprise a group of inherited genetic disorders caused by interruption of normal lymphoid development. These diseases are considered prime candidates for gene therapy by introduction of the missing gene into hematopoietic stem cells… More

DESCRIPTION (provided by applicant): Sleeping Beauty (SB) is a transposon system that has been extensively shown to be capable of mediating integration of new gene sequences into the chromosomes of target cells and tissues. At Discovery Genomics, Inc. (DGI), we are working to develop the SB… More