Clinical Implications of Basic Neuroscience

This review examines data from human and animal studies of the altered huntingtin protein associated with Huntington disease to assess the risk-benefit profile of huntingtin-lowering therapies for the treatment of people with Huntington disease.

This review summarizes findings from studies that suggest that after central nervous system injury, mitochondria can be released into extracellular space as a “help-me signal” to augment recovery.

This review of preclinical applications assesses the potential of the electronic dura mater to deliver electrical and chemical stimulation to targeted areas of the central nervous system for extended periods.

This review discusses the pathogenicity of variants that affect protein levels of TBK1, the gene associated with amyotrophic lateral sclerosis and frontotemporal dementia.

This review assesses the therapeutic potential of the monoclonal antibody to the unique prolyl isomerase Pin1 in the diagnosis and prevention of Alzheimer disease, traumatic brain injury, and chronic traumatic encephalitis.

This study reviews the use of ASOs for the treatment of neurological disorders.

This review article discusses the molecular pathogenic basis for idiopathic generalized epilepsies associated with mutations in the inhibitory GABA_A receptor γ2 subunit gene, GABRG2, an established epilepsy gene.

This article discusses how a new therapeutic system has the potential to augment neuronal and glial signaling and activity for basic and translational applications.

This article discusses putative mechanisms for blood-borne brain rejuvenation and suggests promising avenues for future research and development of therapies.

This article discusses how advances in experimental closed-loop systems hold promise for improved clinical benefit in patients with neurological disorders.

This systematic review examined survival data in elderly patients with glioblastoma being treated with temozolomide therapy alone vs radiotherapy alone.

This study concludes that information on the causative events of Huntington disease might help identify biomarkers that could be used to facilitate clinical trials.

This review article found that the central nervous system inflammatory reaction to brain injury can be deleterious or beneficial, with individual cell populations possessing the ability to be neurotoxic or neuroprotective.

This brief review describes the beneficial functions of endogenous neural stem cells after spinal cord injury.

This review explores a novel nanotherapeutic drug-delivery platform that is mechanically activated within blood vessels by high-fluid shear stresses to selectively target drugs to sites of vascular obstruction.

Turkalp et al review the role of isocitrate dehydrogenase (IDH) in normal physiology and describe aberrations in the IDH pathway that are associated with gliomagenesis, and they review recent work examining the effect of IDH-targeted therapy in cancers harboring IDH mutation and determine how this work has expanded our understanding of the role of IDH in the development and progression of glioma.

A novel analgesic strategy for neuropathic pain is to restore spinal ionotropic inhibition by enhancing KCC2-mediated chloride extrusion. Kahle et al review the data on which this theory is based, discuss high-throughput screens that have searched for small-molecule activators of KCC2, and propose other strategies of KCC2 activation.

Bloom discusses the defining features of Alzheimer disease and the roles amyloid-β and tau play in the disease’s development.

Tassone discusses the inclusion of fragile X syndrome in newborn screening.