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Imagine treating a genetic disease not by managing symptoms for life, but by correcting the underlying genetic error itself. Inventors at NIH are advancing that vision with a gene editing technology designed to deliver highly precise genetic corrections, opening new possibilities for therapies that
The mission of NIMH is to translate the understanding and treatment of mental illnesses through basic and clinical research leading to prevention, recovery and cure. Many pharmaceutical companies generally prefer to invest in more profitable disease areas. NIMH Intramural Research Program (IRP)
Over 300 million people worldwide are suffering from chronic depression. It is also one of the leading causes of disability in US and can be fatal as suicide kills more than 45,000 Americans annually. NIMH psychiatrist Dr. Carlos Zarate and collaborators discovered that a single intravenous dose of
Dr. Wiliam J. Pavan and his team at National Human Genome Research Institute (NHGRI) have established a novel diagnostic tool to predict time of onset for the rare neurodegenerative Niemann-Pick disease type C (NPC). The npc1 gene mutation leads to the lysosomal lipid storage disorder in neuronal