Whitley Vistisen posted an update 3 months, 2 weeks ago
Some of the articles describe a new disease model that enables deeper exploration of key mechanisms.We also present a series of reviews that highlight some of the recent translational advances made in studies of neurodegenerative diseases.These agedependent disorders are becoming increasingly prevalent, in part because the elderly population has increased in recent years. These diseases are diverse in their pathophysiology with some causing memory and cognitive impairments and others reasch Dextrorphan tartrate affecting a persons ability to move, speak and breathe. Effective treatments are desperately needed but will only come with a deep understanding of the causes and mechanisms of each disease.One way to learn about how a disease works is to develop a model system that recapitulates the hallmark characteristics of the disease.Recently, there have been technological innovations that allow for these cells to be cultured in three dimensions, to produce organoids that represent various human tissues, even the brain. These threedimensional brain organoid systems permit cellcell interactions and complex cytoarchitecture to be modeled and studied in greater detail and in more physiological contexts than traditional tissue culture models with isolated cells.Furthermore, accruing evidence suggests that many neurodegenerative diseases are not merely diseases of dying neurons.Nonneuronal cells in the brain, such as glial cells, which are even more abundant in the brain and the central nervous system than neurons, play major roles in disease progression. Incorporating these neuronglial interactions into such D brain organoid models will empower the elucidation of cell nonautonomous disease mechanisms.We anticipate that these D brain organoid systems will be a powerful addition to the disease modelers experimental arsenal.Remarkable advances in genome sequencing technology have made it possible to read genomes of individual patients to discover causes of both rare and common genetic diseases.One highly inspirational success story is the development of a therapy for spinal muscular atrophy. SMA a neuromuscular disease caused by lossoffunction mutations in the SMN gene is the most common genetic killer of babies.Pioneering studies of the molecular mechanisms of the disease and the development of animal models laid the foundation for the recent clinical trials testing antisense oligonucleotides as a therapeutic strategy to correct a splicing defect and restore functional SMN protein.Studies in animal model systems revealed that this therapeutic strategy could work and two recent clinical trials in children with SMA demonstrated that the strategy does work.In a spectacular advance, infants that received the ASO drug showed substantial improvement in motor function compared with children who did not receive the drug. We now have a new hope and a clear path forward for effective therapies for neurodegenerative diseases.It truly is an inspiring and hopeful time to be a researcher in this field.To launch the collection, we present, in this issue, a number of new reviews and research articles that demonstrate the translational impact of studies involving neurodegenerative disease model systems.Below, we summarize the collection so far and provide a preview of whatsto come in upcoming issues of the journal.These insights have also shed light on the complex mechanisms at play in common neurodegenerative diseases.