Most diagnostic exomes on the market today have not been optimized, and have sensitivities of between 20% and 50% for monogenic
Hemophilia, a rare bleeding disorder, is an example of a monogenic
The Institute for Stem Cells in the Treatment and Study of Monogenic
Diseases- is a laboratory which has set out to explore the therapeutic potential of stem cells in the treatment of rare genetic diseases.
These forward-looking statements include, without limitation, references to the versatility, efficiency and safety of ZFN-mediated genome editing, the research and development of novel ZFP TFs and ZFNs and their applications in oncology and the treatment of monogenic
and other rare diseases, partnerships with collaborators and potential clinical trials of ZFP Therapeutics.
This research service identifies key trends in gene therapy for oncology, cardiovascular and monogenic
Analyzes specific renal diseases - both monogenic
disorders confined to the kidney as well as systemic diseases with renal involvement - and explains their genetic causes.
These forward-looking statements include, without limitation, the potential of ZFNs to treat a broad range of human monogenic
diseases, including beta-thalassemia, sickle cell disease, hemophilia A and B, Hurler syndrome, Hunter syndrome, Gaucher disease and other LSDs, research and development of novel ZFP TFs and ZFNs, therapeutic applications of Sangamo's ZFP technology platform, including IVPRP, in indications such as hemophilia and LSDs, the anticipated timing and the number of IND filings, the collaboration with Biogen and initiation and completion of clinical trials.
Lo focused on the future potential for Sequenom's technology to address the unmet needs in detection of monogenic
The wide-ranging array of chapters covers monogenic
and syndromal causes, as well as susceptibility genes; the effects of obesity in the pregnant mother, fetus, children, and old age; and studies of periodic increased weight in animals.
NASDAQ:SQNM), announced that breakthrough data from a collaborative project with The Chinese University of Hong Kong, supporting the noninvasive prenatal diagnosis of monogenic
diseases, were published online today in the Early Edition of the Proceedings of the National Academy of Sciences.
is focused on Engineering Genetic Cures(TM) for monogenic
and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic gene regulation and genome editing.
Drawn from material presented at the Novartis Foundation Symposium entitled "Decoding the Genomic Control of Immune Reactions" held in Canberra in March 2006, this collection covers transcriptional regulatory networks in macrophages, molecular pathways and their role in human disease, specifying the patterns of immune cell migration, human monogenic
disorders and their relationship with specific infections, the genetic control of susceptibility to a strain of tuberculosis, disorders resulting from defective LAT signalosomes, smallpox and mousepox, strategies for phenotype detection and subsequent mapping and cloning, genetic control of host-pathogen interactions, systems genetics, and regulation of the immune system.