Data presented at the American Academy of Neurology (AAN) meeting this week on experimental therapeutics for Huntington’s disease gave some cause for optimism. And, as good research does, they identified new questions as they answered current ones. At the meeting, researchers presented new data on antisense oligonucleotide HTTRx (RG-6042, Ionis Pharmaceuticals Inc./Roche Holding AG) that
Dear Duchenne Community, We want to provide you with an update on the progress of SolidBiosciences IGNITE DMD Phase I/II clinical trial for our investigational microdystrophin gene transfer, SGT-001. Since we communicated preliminary clinical data in February, we advanced the study to evaluate SGT-001 at a higher dose in the second cohort of patients. As
Last year, the birth of the first genetically modified babies shook the world. The German Ethics Council now presents a comprehensive ethical investigation into possible interventions in the genome of human embryos or germ cells. The Council does not deem the human germline to be inviolable. It does, however, consider germline interventions to be ethically
Despite the binary thinking of progressive scientists on the one hand and prophets of doom on the other, science doesn’t tend towards good or evil. Any experiments scientists can do, they will. This is a positive thing, because who is a position to claim omniscience and to forbid it? In fact, it’s human beings who
**For the first time, researchers have developed a non-intrusive way to generate large quantities of stem cells using only a small amount of blood** **The stem cells can repair cells damaged as a result of vascular diseases, which has the potential to prevent blindness and reverse the need for amputations** A discovery by researchers at
The Alliance for Regenerative Medicine (ARM) today released its most recent quarterly data report, offering an in-depth look at cell therapy, gene therapy, tissue engineering, and broader global regenerative medicine sector trends and metrics in the first quarter of 2019. By further curating information provided by ARM’s data partner Informa, the quarterly data report details
A discovery opens the possibility of one day restoring loss of vision by activating the retina’s ability to regenerate. Researchers at Baylor College of Medicine, the Cardiovascular Research Institute and the Texas Heart Institutereveal in the journal Cell Reports that although the mammalian retina – a layer of specialized nerve cells that mediates vision and is located on the back
As a lung transplant surgeon, Matt Bacchetta has watched countless patients wait for an organ, only to not get one. About 80 percent of donor lungs are not in good enough to shape to use. Now Bacchetta and a team of researchers from Columbia University in New York City have shown that they can repair
Regenerative medicine—a field that includes stem cells, bioengineered tissues, and gene therapies—holds great potential. In November 2017, the Food and Drug Administration updated its regulatory framework for these products. Peter Marks, who leads the agency’s Center for Biologics Evaluation and Research (CBER), about the framework gave this interview. Q: What led FDA to update its regulatory framework
In this interview with Bruce Levine Ph.D. (University of Pennsylvania, PA, USA) we find out about his talk on constantly enhancing and improving engineered T cells, which was presented recently at the American Association for Cancer Research (AACR) Annual Meeting (March 29–April 3, 2019, Atlanta, GA, USA). In addition, we also asked about his thoughts
