Translational Neuroscience: Fundamental Approaches for Neurological Disorders
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Translational Neuroscience offers a far-reaching and insightful series of perspectives on the effort to bring potentially revolutionary new classes of therapies to the clinic, thereby transforming the treatment of human nervous system disorders. Great advances in the fields of basic neuroscience, molecular biology, genomics, gene therapy, cell therapy, stem cell biology, information technology, neuro devices, rehabilitation and others over the last 20 years have generated unprecedented opportunities to treat heretofore untreatable disorders of the nervous system. This book provides a wide-ranging yet detailed sample of many of these efforts, together with the methods for pursuing clinical translation and assessing clinical outcomes. Among the topics covered are Alzheimer’s disease, Parkinson’s disease, stroke, multiple sclerosis, epilepsy, motor neuron disease, pain, inborn errors of metabolism, brain tumors, spinal cord injury, neuroprosthetics, rehabilitation and clinical trial design/consideration.
Translational Neuroscience is aimed at basic neuroscientists, translational neuroscientists and clinicians who seek to gain a perspective on the nature and promise of translational therapies in the current era. Both students and established professionals will benefit from the content.
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that are autonomously capable of reversing PD pathology do not always complement one another. Such results need to be taken into consideration before clinical translation of such combinatorial procedures. Human erythropoietin (EPO) enhances the production of red blood cells and is therefore an unlikely candidate for gene therapy of neurodegenerative disorders in CNS. However, recent research has indicated strong neuroprotection of dopaminergic neurons achieved by EPO expression in the brain.
(typically 20 mer), modiﬁed DNA-like chemicals that bind to a speciﬁc RNA target sequence by typical Watson–Crick base pairing (reviewed in ). The chemical modiﬁcations of the ASO increase its stability in biologic ﬂuids, increase the potency of binding to the target, and particular modiﬁcations of the ASO inﬂuence the effect of the ASO-RNA duplex in the cell. A typical ASO modiﬁed at the 2′ position on the ﬁrst 5 and last 5 nucleotides without modiﬁcations in the center 10 nucleotides will
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