monogenic disease


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Related to monogenic disease: Genetic diseases, autosomal dominant disorders, single gene disorder
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Synonyms for monogenic disease

References in periodicals archive ?
Lo focused on the future potential for Sequenom's technology to address the unmet needs in detection of monogenic diseases.
These forward-looking statements include, without limitation, references to the research and development of novel ZFNs, potential therapeutic applications of the ZFN technology for the treatment of hemophilias and other monogenic diseases and potential milestone payments.
Sangamo's IVPRP is designed to provide a one-time, potentially curative treatment for patients with monogenic diseases, such as hemophilia and LSDs, who currently require repeated infusions of protein replacement therapies throughout their lives.
According to the World Health Organization, monogenic diseases result from modifications in a single gene occurring in all cells of the body.
These forward-looking statements include, without limitation, references relating to expected timing for initiating clinical trials, filing of INDs and release of clinical trial data, research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform, the potential of Sangamo's ZFP technology to treat HIV/AIDS and a variety of monogenic diseases including Hunter's disease, Hurler's disease and other LSDs, hemophilia A and B, and Huntington's disease, and the ability to expand the ZFP Therapeutic pipeline.
These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform; the potential of Sangamo's ZFP technology to treat LSDs, Hunter, Hurler and Gaucher syndromes and the safety of the approach of using ZFP-mediated genome editing in vivo; the expected IND studies for Hunter and Hurler programs; and the applicability of Sangamo's ZFP technology in monogenic diseases.
The Company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington's disease, and other monogenic diseases, and with Biogen Idec for hemoglobinopathies.
Sangamo is also developing sequence-specific ZFP-Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases such as sickle cell anemia and for infectious diseases such as HIV.
Twelve oral and six poster presentations, given by Sangamo scientists and their academic collaborators, will detail data from Sangamo's therapeutic and research programs including HIV/AIDS, hemoglobinopathies, lysosomal storage disorders, and other monogenic diseases, cancer immunotherapy and advancements in technology including delivery.
Other therapeutic development programs are focused on macular degeneration, ischemic heart disease, congestive heart failure, neuropathic pain, and infectious and monogenic diseases.