A cutting-edge gene therapy has significantly restored hearing in children and adults with congenital deafness, showing dramatic results just one month after a single injection. Researchers used a virus to deliver a healthy copy of the OTOF gene into the inner ear, improving auditory function across all ten participants in the study. The therapy worked best in young children but still benefited adults, with one 7-year-old girl regaining almost full hearing. Even more exciting: this is just the start, as scientists now aim to target other genes that cause more common forms of deafness.
A new study has revealed that chemical tags once regarded as genetic clutter are in fact powerful gene silencers – and removing them could unlock safer treatments for inherited blood disorders.
Advances in technology have led to the miniaturization of many mechanical, electronic, chemical and biomedical products, and with that, an evolution in the way these tiny components and parts are transported is necessary to follow. Transport systems, such as those based on conveyor belts, suffer from the challenge of friction, which drastically slows the speed and precision of small transport.
Researchers from Yokohama National University addressed this issue by developing an untethered levitation device capable of moving in all directions. The frictionless design allows for ultrafast, agile movement that can prove to be very valuable in machine assembly, biomedical and chemical applications via contactless transport.
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Take note of the name: ReHMGB1. A new study pinpoints this protein as being able to spread the wear and tear that comes with time as it quietly travels through the bloodstream. This adds significantly to our understanding of aging.
Short for reduced high mobility group box 1, ReHMGB1 triggers senescence in cells, permanently disabling them. It doesn’t just do this locally; it can send damaging signals throughout the body, particularly in response to injuries or disease.
“An important question in aging research is why senescent cells increase with age,” write the study authors, led by researchers from the Korea University College of Medicine.
A new study, led by researchers at Children’s Hospital of Philadelphia (CHOP), identified tiny pieces of messenger RNA that are missing in pediatric high-grade glioma tumors but not in normal brain tissues. Preclinical research indicates that these missing RNA fragments can make difficult-to-treat tumors more responsive to immunotherapy. The findings were recently published in the journal Cell Reports.
One of the biggest challenges facing cancer research is the need to find safe and effective therapies for the most aggressive types of brain tumors. Adoptive immunotherapies with CAR-T cells are promising; however, they often also target healthy cells, which share most surface proteins with cancerous cells. While this collateral damage might be tolerable in patients with certain types of blood cancer, in the brain, wiping out healthy neurons is unacceptable. This means that deep knowledge of gene expression patterns exclusive to tumor cells is critical.
A potential means of discovering new therapeutic targets for brain tumors may lie in alternative splicing, a process whereby a single gene produces multiple proteins by rearranging exons, the building blocks of messenger RNA, in different combinations. Researchers suspected that splicing in glioma cells may differ from splicing in normal brain cells, which could help devise new therapeutic interventions.
Synthetic cells are artificial constructs designed to mimic cellular functions, offering insights into fundamental biology, as well as promising impact in the fields of medicine, biotechnology, and bioengineering. In this perspective, the authors highlight major scientific hurdles, such as the integration of functional modules by ensuring compatibility across diverse synthetic subsystems, and propose strategies to advance the field.
Researchers at the Institute for Bioengineering of Catalonia (IBEC) in collaboration with the Dexeus University Hospital have captured unparalleled images of a human embryo implanting. This is the first time that the process has been recorded in real time and in 3D.
Hospital Clínico San Carlos in Madrid-led research reports that intermittent theta-burst transcranial magnetic stimulation (TMS) paired with language therapy over six months was associated with positive outcomes in primary progressive aphasia (PPA). Improvements included less decline in regional brain metabolism and improvements in language abilities, functional independence, and neuropsychiatric symptoms.
Primary progressive aphasia is a neurodegenerative clinical syndrome with insidious onset characterized by prominent speech and/or language impairment. It is a syndrome that can be the mode in which common causes of dementia, Alzheimer’s disease and frontotemporal degeneration are initially present.
According to current international consensus criteria, three variants are recognized: nonfluent/agrammatic, semantic, and logopenic. Speech-language intervention has proven to be beneficial.