Drugs that manipulate the code of life are finally changing lives Source Scientific American The idea for gene therapy—a type of DNA-based medicine that inserts a healthy gene into cells to replace a mutated, disease-causing variant—was first published in 1972. After decades of disputed results, treatment failures and some deaths in experimental trials, the first
The strategy has long been seen as having enormous potential to cure diseases Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing. The feat, published in the journal Nature Biotechnology, offers
Mutations in the RIPK1 gene responsible for CRIA syndrome. Over the last 20 years, three families have been unsuspectingly linked by an unknown illness. Researchers at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and other organizations have now identified the cause of the illness, a new disease called
Potential 1st Gene Therapy in Europe Directed at Any Type of Hemophilia Application to be Reviewed Under Accelerated Assessment BioMarin Pharmaceutical announced today that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January
A research team at Nanyang Technological University, Singapore (NTU Singapore) has found an easier way to harvest healing factors – molecules that promote tissue growth and regeneration – from adult stem cells Presently, scientists ‘pre-condition’ adult stem cells to secrete healing factors by putting them in a low-oxygen chamber or by using biochemicals or genetic engineering.However in
Tissue engineering and regenerative medicine (TERM) are paving the way to the generation of functional and mature biological tissues that closely emulate cellular, biochemical, and mechanical cues. Electrical fields in the human body modulate myriad biological processes, such as synapses, muscle contraction, hearing, and wound healing, which were disregarded in TERM until recently. The human
Human platelet lysate (HPL), rich in growth factors, is an efficient alternative supplement to fetal bovine serum (FBS) for ex vivo propagation of stromal cell-based medicinal products. Since 2014, HPL has been a focus of the Working Party for Cellular Therapies of the International Society of Blood Transfusion (ISBT). Currently, as several Good Manufacturing Practice
Researchers at Ohio State University and the University of Texas Southwestern Medical Center say they have developed a new gene therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes. Though the work was intended as a proof of concept,
– Approval supported by data from the pivotal Phase 3 SELECT rheumatoid arthritis program evaluating nearly 4,400 patients– In five pivotal Phase 3 studies, RINVOQ™(upadacitinib) met all primary and ranked secondary endpoints across a variety of adult patient populations with moderate to severe active rheumatoid arthritis– RINVOQ, given alone or with csDMARDs, demonstrated improved rates
A team of researchers from the University of Georgia’s Regenerative Bioscience Center has found that neural exosomes—“cargo” molecules within the nervous system that carry messages to the brain—can minimize or even avert progression of traumatic brain injury when used as part of a new cell-to-cell messaging technology. The finding could result in the first delivery platform and
