Not exact matches
As he was working to
identify a profitable
research direction, his thoughts «all crystallized around
rare diseases.»
«Steep funding cuts for the federal health agencies are counterproductive at a time when innovative
research is moving us closer to
identifying solutions for
rare diseases, new prevention strategies to protect Americans from deadly and costly conditions, advances in gene therapy, new technologies for understanding the brain, and treatments that harness the ability of our immune system to fight cancer.»
Our
research has
identified a new gene, which is the cause of a severe,
rare human
disease.
Building on
research that
identified a
rare genetic mutation in Italian people that leads to the early onset of Alzheimer's and one in Icelandic people that delays the onset of the condition, a researcher at the University of British Columbia has discovered that using an enzymatic scissor the right way could stave off the cognitive decline associated with the
disease.
The strategy developed by
research teams and development of I - Stem aims to
identify innovative therapies applicable to
rare genetic
diseases based on exploring the potential offered by human pluripotent stem cells.
St. Jude Children's
Research Hospital scientists studying two
rare, inherited childhood neurodegenerative disorders have
identified a new, possibly common source of DNA damage that may play a role in other neurodegenerative
diseases, cancer and aging.
However, a great deal of basic
research is still required to understand the molecular bases of
rare diseases and
identify potential therapeutic targets.
The Broad Institute and its collaborating institutions have received two major grants from the National Human Genome
Research Institute (NHGRI) that will support the use of genome sequencing and analysis to
identify the genes and genetic variation that underlie both
rare and common
diseases.