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I.10 Gene therapy and other novel treatment approaches for CMT
There is still no effective drug treatment available for Charcot-Marie-Tooth disease (CMT). Current management relies on rehabilitation therapy, surgery for skeletal deformities, and symptomatic treatment. The challenge is to find disease-modifying therapies. Several approaches, including gene silencing (by means of ASO, siRNA, CRISPR-Cas9 editing) to counteract the PMP22 gene overexpression in the most frequent CMT1A type are under investigation. PXT3003 is the compound in the most advanced phase for CMT1A, as a second phase-III trial is ongoing.
Source: Neuromuscular Disorders - October 1, 2022 Category: Neurology Authors: D. Pareyson Source Type: research

Advanced molecular therapies for neurological diseases: focus on stroke, alzheimer's disease, and parkinson's disease
AbstractNeurological diseases (NDs) are one of the leading causes of disability and the second leading cause of death globally. Among these stroke, Alzheimer's disease (AD), and Parkinson's disease (PD) are the most common NDs. A rise in the absolute number of individuals affected with these diseases indicates that the current treatment strategies in management and prevention of these debilitating diseases are not effective sufficiently. Therefore, novel treatment strategies are being explored to cure these diseases by addressing the causative mechanisms at the molecular level. Advanced therapies like gene therapy (gene ed...
Source: Neurological Sciences - September 6, 2022 Category: Neurology Source Type: research

Principles of translational gene therapy for neuromuscular diseases
This article describes the principles of precision gene therapy for neurogenetic diseases using examples of neuromuscular diseases.DISCUSSION: Various strategies of gene therapy have become established and are being tested in preclinical and clinical trials and evaluated as approved forms for long-term efficacy. The aim of every gene therapy is the modification or introduction of the target gene with initiation of a degradation of dysfunctional proteins. Various techniques, such as gene transfer, gene substitution or gene editing in vivo and ex vivo are now usable. For example, a modification of the pre-mRNA using antisens...
Source: Der Nervenarzt - April 6, 2022 Category: Neurology Authors: B Schoser Source Type: research

PEG-PEI/siROCK2 inhibits Aβ42-induced microglial inflammation via NLRP3/caspase 1 pathway
Conclusion PPSR inhibits Aβ42-induced microglial inflammation via NLRP3/caspase 1 pathway.
Source: NeuroReport - December 10, 2021 Category: Neurology Tags: Cellular, Molecular and Developmental Neuroscience Source Type: research

Sprouty2 —a Novel Therapeutic Target in the Nervous System?
AbstractClinical trials applying growth factors to alleviate symptoms of patients with neurological disorders have largely been unsuccessful in the past. As an alternative approach, growth factor receptors or components of their signal transduction machinery may be targeted directly. In recent years, the search for intracellular signaling integrator downstream of receptor tyrosine kinases provided valuable novel substrates. Among them are the Sprouty proteins which mainly act as inhibitors of growth factor-dependent neuronal and glial signaling pathways. In this review, we summarize the role of Sprouties in the lesioned ce...
Source: Molecular Neurobiology - May 7, 2019 Category: Neurology Source Type: research