Novo Nordisk has moved
into sickle cell disease through a licensing deal (PDF) with EpiDestiny.
Finally, the researchers injected these partially differentiated cells
into sickle cell mice that had been treated with radiation to kill their own blood stem cells.
Not exact matches
Government funding for research
into sickle - cell disease is pitifully inadequate (the Sickle - Cell Society receives no government funds at
sickle -
cell disease is pitifully inadequate (the Sickle - Cell Society receives no government funds at a
cell disease is pitifully inadequate (the
Sickle - Cell Society receives no government funds at
Sickle -
Cell Society receives no government funds at a
Cell Society receives no government funds at all).
It occurs when some of the red blood
cells change
into a «
sickle» shape and cause a buildup of red blood
cells in small blood vessels.
This elusive stability must be achieved before stem
cell supplies can be kept on hand until it is time to turn them
into replacements for say misshapen red blood
cells seen in
sickle cell anemia or abnormal white blood
cells causing leukemia.
For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights
into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and test a «curative therapy» for the first molecular disease:
sickle cell disease.
Then we corrected the
sickle -
cell mutations in those
cells and injected them back
into the diseased mice.
The anti-
sickling HSC will be transplanted back
into the patient's bone marrow and multiply the corrected
cells that make red blood
cells without
sickling.
Rod Kellems, Ph.D., study co-author and chairman of the Department of Biochemistry and Molecular Biology at the UTHealth Medical School, added, «This research provides insight
into how red blood
cells work, revealing that SphK1 - mediated elevation of S1P contributes to
sickling and promotes disease progression and highlights potential therapeutic opportunities for
sickle cell disease.»
Scientists have used the disease - in - a-dish strategy to gain insight
into sickle -
cell anemia and heart arrhythmias.
In a landmark study that could lead to new therapies for
sickle cell anaemia and other blood disorders, UNSW Sydney - led researchers have used CRISPR - gene editing to introduce beneficial natural mutations
into blood
cells to boost their production of Read more about Solution to 50 - year - old mystery could lead to gene therapy for common blood disorders - Scimex
Stem
cell researchers are getting closer to a new treatment for
sickle cell disease, moving promising laboratory research
into human clinical trials.
One early form of CRISPR - based gene therapy could involve editing the genes responsible for blood disorders like
sickle -
cell anemia in bone marrow
cells, growing them
into mature blood
cells and injecting them back
into patients.
Salk researchers reprogrammed skins
cells taken from a
sickle cell disease patient
into induced pluripotent stem
cells (iPSCs), immature
cells capable of developing
into any type of bodily tissue.
This booklet has been produced based on research
into young people with
sickle cell disorder in schools in England.
The National Center on Birth Defects and Developmental Disabilities recently translated key
sickle cell fact sheets
into Spanish and French.