Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their
The FDA has granted orphan drug designation to ADP-A2M4, an investigational specific peptide-enhanced affinity receptor T-cell therapy, for the treatment of soft tissue sarcomas. ADP-A2M4 (Adaptimmune) is an autologous, genetically modified SPEAR T-cell therapy that targets the MAGE-A4 antigen on the surface of cancer cells. Certain soft tissue sarcomas express MAGE-A4 at high levels and
Dogs with a Crohn’s-like disease fed a special diet were found to have characteristic changes in their gut microbiomes, paralleling changes seen in children with Crohn’s. uch remains mysterious about the factors influencing human inflammatory bowel disease (IBD), but one aspect that has emerged as a key contributor is the gut microbiome, the collection of
An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients’ sight. But new research underscores the importance of further investigation to halt the progression of the disorder. Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was
In pair of studies, UCLA researchers describe new ways to treat a metabolic liver disorder Source UCLA Newsroom Most babies who are born with arginase deficiency — a rare genetic disease that leads to the accumulation of the amino acid arginine in the blood — don’t have symptoms at first. By the time they’re toddlers,
NIH-funded research project develops new method to preserve human livers for transplantation. Source NIH Scientists have greatly extended the amount of time human livers can be stored for transplantation by modifying a previous protocol to extend the viability of rat livers. Previously, human livers were only viable for an average of nine hours, but the
Source https://www.liebertpub.com/doi/full/10.1089/ten.teb.2019.0047#utm_source=FastTrack&utm_medium=email&utm_campaign=teb Bone regeneration procedures in clinics and bone tissue engineering stand on three pillars: osteoconduction, osteoinduction, and stem cells. In the last two decades, the focus in this field has been on osteoinduction, which is realized by the use of bone morphogenetic proteins and the application of mesenchymal stem cells to treat bone defects.
Stem cell company TreeFrog Therapeutics has received close to €3 million in non-dilutive funding from French and European programs. The company received €450,000 from the French government as part of Phase 2 of the i-Nov Innovation Challenge, as well as €2.4 million from the European Commission as part of Phase 2 of the SME Instrument.
A Chicago-based center that has long operated a clinical trial program for stem cell therapies, has stopped recruiting further patients as its chief, Richard Burt, leaves for a research sabbatical. Source The Scientist A pioneering stem cell clinical trial program in the US is closing up shop, a surprise move for what had appeared to
Private clinics offering tantalising hopes from stem cell treatment are being accused of taking advantage of desperate patients and exposing them to risks. New Zealand’s public health system has offered stem cell bone marrow transplants for cancer patients for many years, but a collection of private clinics have also sprung up offering expensive stem cell
