Scripps Research Institute scientists have offered new hope for parents whose children suffer from the rare genetic disorder ‘cystinosis’ by showing through an experiment on mice that stem cell transplantation can successfully correct the defect (…)
In the study, the researchers used bone marrow stem cell transplantation to address symptoms of cystinosis in a mouse model (…)
In the new study, the researchers found that transplanted bone marrow stem cells carrying the normal lysosomal cystine transporter gene abundantly engrafted into every tissue of the experimental mice (…)
“The results really surprised and encouraged us. Because the defect is present in every cell
Scientists and transplant clinicians at the Ansary Stem Cell Institute at Weill Cornell Medical College and the Center for Cell Engineering at Memorial Sloan Kettering Cancer Center have been awarded a $15.7 million, four-year research grant from the New York State Stem Cell Science Program (NYSTEM).
The scientists will translate their approach to manipulate hematopoietic stem cells to cure acquired and inherited blood disorders. For many patients with such blood diseases, including sickle cell disease, the only hope for a cure requires transplanting normal blood stem cells.
But in many instances suitable blood stem cells cannot be found or there are
Novogen Limited, an oncology drug development company, today announced a collaboration with Australian company, Genea Biocells, to accelerate testing of their super-benzopyran (SBP) drugs for degenerative diseases of the nervous system and muscles.
Preliminary research conducted by both companies found that super-benzopyrans appear to be effective at promoting the ‘normalization’ of stem cells associated with some forms of neurodegeneration and muscular dystrophy, genetic disorders which result in progressive deterioration of brain function or muscle strength and function.
“SBPs have already been shown to be highly effective at killing cancer stem cells, which were previously considered resistant to anti-cancer therapy.
But in some
The prospect of treating genetic diseases with corrected stem cells grown from patients’ own bodies has moved closer, after the results of a remarkable experiment.
Scientists have successfully reprogrammed skin tissue from people with a rare form of anaemia to create powerful stem cells, while at the same time rectifying the genetic defect that causes the condition.
The corrected stem cells could be grown into blood precursor cells for therapy. As these would carry a patient’s own DNA, except for the mutation responsible for the illness, they could be transplanted without risk of rejection by the body’s immune system.
Though the research
Image by thomtrance via Flickr
An experiment successfully performed by researchers at the University of Wisconsin-Madison and the University of Missouri in Colombia, described in Nature magazine shows that cells removed from a patient’s skin and transformed into cells similar to embryonic stem cells have become a laboratory model for diseases and can be observed in real time and studied to find new cures. The researchers recreated spinal muscular atrophy (SMA) pluripotent stem cells removed from the skin of a child affected by the neurodegenerative genetic disease. In the laboratory, the cells behaved exactly as they do in