Pre-marital counselling and screening is one method of decreasing the likelihood of children being born with monogenic
The reason this is relevant for Qatar is that the structure of the society encourages a high degree of consanguineous marriage, so the frequency of these monogenic
diseases is quite high.
In the former case, discovery efforts continue globally, and over time we will understand the primary genetic lesions that cause all monogenic
We are delighted to be partnering the first of our monogenic
disease programs with Shire, a company known for its development of innovative medicines for genetic diseases," said Edward Lanphier, Sangamo's president and chief executive officer.
Sangamo's other therapeutic programs are focused on monogenic
diseases, including hemophilia and hemoglobinopathies such as sickle cell anemia and beta-thalassemia, and a program in Huntington's disease.
Partnership will focus on reproductive health, new born screening, autism, hereditary cancer, monogenic
disorders, and exome sequence-based personalised medical healthcare.
, and polygenic causes of stroke are examined, and risk factors such as oral contraceptives, migraine, and drug abuse are examined.
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.
This delivery approach achieved efficiencies of gene transfer and targeted integration of the therapeutic DNA sequence, particularly in more primitive longer-lasting stem cells and T-cells, to levels that can be used therapeutically to potentially treat a range of monogenic
Researchers in Europe and the US report on their work, with discussion of genome architecture, clinical evaluation, database aids, 3D shape and molecular analyses of facial dysmorphology, monogenic
causes, and the microdeletion/duplication syndromes, among other topics.
Trends in clinical development have shown that there is significant focus on terminal diseases such as monogenic
and oncology disorders," notes Mr.
In many instances the renal disease is one component of a complicated systemic hereditary disease, either monogenic
Sangamo's hemophilia B program is the first therapeutic application of its IVPRP strategy, an in vivo targeted integration strategy that can be leveraged across multiple monogenic
diseases that are currently treated using protein or enzyme replacement therapy.
Geneticists, cardiologists, and other specialists review the scientific findings over the past few years about four monogenic
disorders that all turn out to be caused by mutations that alter the function of molecules in a common signaling cascade, the Ras-MAPK pathway.
Future Applications in Detection of Monogenic