monogenic disease


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

monogenic disease

A disease that is inherited from one generation to the next in classic mendelian fashion.
See also: disease
References in periodicals archive ?
NASDAQ: SGMO), a leader in genome-editing technology, announced today that they have entered into a collaboration and license agreement to develop therapeutics for hemophilia and other monogenic diseases based on Sangamo's zinc finger DNA-binding protein (ZFP) technology.
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 for the treatment of hemophilia B and other monogenic diseases, the timing of submissions of INDs for the treatment of hemophilia B, hemophilia A and lysosomal storage diseases, and the timing of submissions of INDs for other partnered programs and Sangamo proprietary programs.
Prenatal diagnosis of monogenic diseases is currently performed by invasive procedures such as amniocentesis and chorionic villus sampling (CVS) which carry a risk of fetal miscarriage.
These data highlight that our proprietary ZFN-enabled genome editing technology can generate broadly applicable and disruptive approaches for the potential cure of numerous monogenic diseases including LSDs," stated Edward Lanphier, Sangamo's president and CEO.
These new data significantly 'de-risk' Sangamo's approach for Hunter and Hurler syndromes and potentially many other monogenic diseases, such as the hemophilias, that are currently treated using lifelong enzyme replacement therapy," stated Edward Lanphier, Sangamo's president and chief executive officer.
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 Inc.
In order to exploit fully these potentials of hES cell lines within the framework of its exploration of therapeutics for monogenic diseases, I-STEM has set up a screening department through a close partnership with the companies Velocity11, Discngine and Prestwick Chemical.