Researchers find animal model for understudied type of muscular dystrophy

Researchers find animal model for understudied type of muscular dystrophy
As researchers develop drugs that target the DUX4 protein, the hope is that these mice will be used to determine whether such drugs can reach skeletal muscle and allow muscle damage to be repaired, even in the presence of DUX4. The mouse model designed by Kyba and his team allows the disease-associated DUX4 protein to be produced when mice are treated with doxycycline. The amount of DUX4 can be controlled...

Biologists reprogram skin cells to mimic rare disease

Biologists reprogram skin cells to mimic rare disease
Finally, the investigators used their regimen on skin cells from patients with familial dysautonomia, then compared these familial dysautonomia-neural crest cells to the control neural crest cells made from healthy adults. They identified 412 genes with lower activity levels in the familial dysautonomia-neural crest cells, of which 98 are involved in processing RNA products made from active genes. The...

Tissue development ‘roadmap’ created to guide stem cell medicine

Tissue development 'roadmap' created to guide stem cell medicine
The two papers also clarify uncertainty around which methods are best for stem cell engineering, and should advance the use of cells derived from patient tissues to model disease, test potential drugs and use as treatments. For example, using CellNet, one of the studies unexpectedly found that skin cells can be converted into intestinal cells that were able to reverse colitis in a mouse model. CellNet...

Clinical trial evaluates safety of stem cell transplantation in spine

Clinical trial evaluates safety of stem cell transplantation in spine
Pre-clinical studies of these cells by Ciacci and Martin Marsala, MD, at the UC San Diego School of Medicine, showed that these grafted neural stem cells improved motor function in spinal cord injured rats with minimal side effects indicating that human clinical trials are now warranted. All participants will receive the stem cell injection. The scientists will use a line of human stem cells approved...

How breast cancer usurps powers of mammary stem cells

How breast cancer usurps powers of mammary stem cells
Story Source: During pregnancy, a new mammary stem cell population arises, distinct from those involved in development and maintenance of the non-pregnant gland. These stem cells remodel the breasts and lactating glands in preparation for feeding the newborn child. Normally, these stem cells contribute only to early remodeling events and are switched off by the time milk production begins. The above...

Growing human GI cells may lead to personalized treatments

Growing human GI cells may lead to personalized treatments
“An additional important feature of this system is that we can isolate stem cell lines from intestinal biopsies,” said first author Kelli L. VanDussen, PhD, a postdoctoral fellow in Stappenbeck’s laboratory. “These biopsies are very small tissue fragments that are routinely collected by a gastroenterologist during endoscopy procedures. We have refined this technique, so we have...

Breakthroughs made in ovarian cancer research

Breakthroughs made in ovarian cancer research
By applying bioinformatics analysis on big cancer genomics data[5], BII scientists were able to identify genes whose mutation status could be used for prognosis and development of personalized treatment for HG-SOC. Prof Sir David Lane, Chief Scientist, A*STAR, said, “These findings show how the various research institutes at A*STAR offer their expertise in developing new approaches to examine...

Stem cells show promise for stroke in pilot study

Stem cells show promise for stroke in pilot study
Although the trial was mainly designed to assess the safety and tolerability of the treatment, the patients all showed improvements in their condition in clinical tests over a six-month follow-up period. A bone marrow sample was taken from each patient. The CD34+ cells were isolated from the sample and then infused into an artery that supplies the brain. No previous trial has selectively used CD34+...

Another potential ALS treatment avenue identfied by researchers

Another potential ALS treatment avenue identfied by researchers
This latest finding is expected to push towards clinical studies the second major ALS discovery from Eggan’s lab in less than a year. The HSCI stem cell biologist, and his neuroscience and neurology collaborators at Massachusetts General Hospital and Boston Children’s Hospital, are preparing for a phase I clinical trial of a medication already approved for epilepsy which Eggan and colleagues...

Seamless gene correction of beta-thalassemia mutations in patient-specific cells

Seamless gene correction of beta-thalassemia mutations in patient-specific cells
?-thalassemia results from inherited DNA mutations in the hemoglobin beta (HBB) gene, resulting in reduced HBB expression in red blood cells and, in the most severe forms, anemia. The only established curative treatment is hematopoietic stem cell transplantation; however, this treatment requires a matched donor. Gene therapy, which delivers a corrected copy of a gene into patient cells, could bypass...