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Motor neuron diseases (MNDs) comprise a large and heterogeneous group of disorders in which the impairment of neuromuscular unity is the major pathological hallmark, causing severe morbidity to individuals, and frequently leading to death due to respiratory failure. The incidence of MNDs varies in different populations; however, the most prevalent, Amyotrophic Lateral Sclerosis, is estimated to occur globally in around 1/2 per 100 000. As of today, a myriad of pathogenic variants located at more than a hundred genes and loci have been associated with this complex group of entities. Functional studies aiming to understand their physiological impact, both using stem cell and animal models, hav...
While most stem cell books focus on basic aspects and/or cell therapy, this book emphasizes the relevance of stem cells obtained from patients, the so-called “patients in a petri dish” as tools to investigate human genetic diseases for which there are no available effective treatment. Chapters embrace several examples of the use of iPS cell technology, a recent Nobel Prize-winning scientific breakthrough, to obtain patient-specific pluripotent cells from which many types of specialized cells involved in a particular disease can be generated, including psychiatric and neurodegenerative disorders, muscular dystrophies, laminopathies, among others. The text is a current and timely resource for postgraduate students, scientists and clinicians, interested in applications of this rapidly developing field of research in disease modeling, drug development, and emerging issues that it brings to regenerative medicine.
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RNA enzymes based on the R3C ligase ribozyme motif were previously shown to undergo both self- and cross-catalytic replication, albeit very inefficiently. In an attempt to develop efficient cross-replicating RNA enzymes that can undergo self-sustained exponential amplification in the absence of proteins, in vitro evolution was carried out to optimize their catalytic activity. Two enzymes, E and E, ́ were selected for their ability to catalyze each other's synthesis from a total of four component substrates (A, B and A, ́ B)́. The optimized cross-replicating RNA enzymes were able to undergo self-sustained exponential amplification at a constant temperature in the absence of proteins or oth...
It has become evident over the last years that abnormalities in RNA processing play a fundamental part in the pathogenesis of neurodegenerative diseases. Cellular viability depends on proper regulation of RNA metabolism and subsequent protein synthesis, which requires the interplay of many processes including transcription, pre--‐mRNA splicing, mRNA editing as well as mRNA stability, transport and translation. Dysfunction in any of these processes, often caused by mutations in the coding and non--‐ coding RNAs, can be very destructive to the cellular environment and consequently impair neural viability. The result of this RNA toxicity can lead to a toxic gain of function or a loss of fun...
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Investigating in the area of perovskite materials and the fabrication of devices for properties in optoelectronics, we have presented a brief outline of perovskite materials. The authors present a fairly comprehensive arrangement of this very active area of research, with its past changes and present position and outlooks. Discussions are presented regarding photocatalysis, fabrication of solar cell devices and their stability, lead-free materials, as well as thermoelectric and piezoelectric applications. In view of the present status of perovskite materials, I am assured that each chapter of the book will be of boundless encouragement for researchers, scientists, and academicians working in this field.
Showcasing the expertise of top-tier specialists who contributed to the newly released guidelines for the care of thrombosis in cancer patients, this exciting guide was written and edited by members of the American Society of Clinical Oncology panel, (ASCO), on the prevention and treatment of cancer-associated thrombosis, among others, and provides