Original story from the International Society for Stem Cell Research The ISSCR is updating its Guidelines for Stem Cell Research and Clinical Translation to respond to recent scientific advances that include the use of pluripotent stem cell (PSC) to create models of early human embryo development. As the science continues to advance, it raises important
Researchers investigating childhood leukaemia have discovered that increasing MLL gene expression in iPSCs drives hematopoietic stem cell production, so could be the target of new therapies. Researchers have identified that increasing the expression of the normal form of the MLL gene can drive production of hematopoietic stem cells (HSCs). This not only provides a protocol
Source World Economic Forum The current market for stem cell therapies is growing at 36% per year and will rapidly expand when a breakthrough treatment for non-communicable disease or a lifestyle factor occurs. Funding is flowing into the sector from venture capitalists and large companies. With greater resources and an increasingly experienced talent pool, scientists
The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials.
First agreements with statutory health insurances utilize bluebird’s innovative value-based payment model and provide coverage for ZYNTEGLO for up to 50% of patients in Germany First qualified treatment center established at University Hospital of Heidelberg to provide ZYNTEGLO to patients bluebird bio, Inc. announced the launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a
ARM today announced the release of a report, “A Transformative Therapy Value Model for Rare Blood Diseases.” The report uses a refined value model to assess the impact of durable and potentially curative cell and gene therapies on health system costs. Produced in collaboration with the Marwood Institute, this report calculates the potential cost savings that
Johns Hopkins researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a “suicide gene” to pediatric brain tumor cells implanted in the brains of mice. The poly(beta-amino ester) nanoparticles, known as PBAEs, were part of a treatment that also used a drug to kill the cells and prolong the test
Scientists have discovered how to switch off a key ‘pain gene’, dramatically raising hopes of a long-term treatment to relieve the agony of serious illness for millions. The revolutionary technique alters a patient’s DNA, silencing a gene that transmits pain signals up the spine. Preliminary studies on mice have already proven successful and US researchers
Researchers are moving closer to a new approach for improving spinal fusion procedures and repairing broken or defective bones that avoids an over-production of bone that commonly occurs in current treatments. In a preclinical study, researchers significantly reduced undesired bone growth outside of targeted repair areas in rat femurs by delivering a potent bone-forming protein
The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many
