Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the new technology can be likened to dramatically accelerating evolution from millions of years to weeks.
The FDA unexpectedly reversed course Thursday, approving the second treatment developed by Sarepta Therapeutics for the rare disease Duchenne muscular dystrophy just a few months after rejecting the drug. Sarepta announced Thursday evening that the agency had approved its second drug, called Vyondys 53. After the drug was originally rejected by the FDA in August due to concerns over the
Advance could help identify drugs to eventually treat fibrosis Every organ in the body is capable, to some extent, of repairing itself after an injury. As part of this process, scar tissue forms and then recedes to make room for normal tissue when healing is complete. However, when healing is disrupted — whether by chronic
Organoids have extensive therapeutic potential and are increasingly opening up new avenues within regenerative medicine. Source Nature Communications Their clinical application is greatly limited by the lack of effective GMP-compliant systems for organoid expansion in culture. Here, it’s envisaged that the use of extracellular matrix (ECM) hydrogels derived from decellularized tissues (DT) can provide an
Findings from the ABC-PICU study may alter policies at hospitals where fresh red cells are preferentially used A new study led by the Sainte-Justine University Hospital Centre and the University of Montreal, and Washington University School of Medicine in St. Louis has found that transfusions using fresh red blood cells—cells that have spent seven days
Hematopoietic stem cells (HSCs) are responsible for the constant replenishment of all blood cells throughout life. One of the major challenges in regenerative medicine is to produce tailor-made HSCs to replace the defective ones in patients suffering from blood related diseases. This would circumvent the shortage of donor HSCs available for the clinic. To achieve
Led by researchers at Baylor College of Medicine, a study published in the journal Cell Stem Cell reveals a new mechanism that contributes to adult bone maintenance and repair and opens the possibility of developing therapeutic strategies for improving bone healing. “Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized,”
Scientists from The Australian National University (ANU) have invented a new jelly material that mimics biological matter such as skin, ligaments and bone, and which is very strong, self-healing and able to change shape. The team say the hydrogel could enable a new class of medical implants or artificial muscles for next-generation robots that could one
The International Society for Cell and Gene Therapy (ISCT) has formed a consortium to tackle what it says is a rising number of unscrupulous and unproven cell banking players. With the rise of interest in the cell and gene therapy sector, industry and the market have been plagued with unproven products and services from rogue
The first two patients treated with the 3e13 vg/kg dose achieved stable Factor VIII (FVIII) levels demonstrating durability in the normal range through 44 and 37 weeks, respectively– All five patients in the 3e13 vg/kg dose cohort achieved normal range FVIII levels within 5-7 weeks following treatment, with no bleeding events with up to 44 weeks
