Abstract:
PURPOSE OF REVIEW:Most patients suffering from muscular dystrophies can now obtain a precise diagnosis of their underlying molecular defect, but no efficient treatment to prevent disability and death. This review summarizes recent progress towards developing efficient treatments for these severe diseases. RECENT FINDINGS:Different levels of progress have been achieved in three main approaches: gene therapy, cell therapy and pharmacological therapy. Gene therapy has progressed by improving different vectors for gene delivery. Adenoviruses (mainly high capacity versions) and adeno-associated viruses were the most explored viral vectors. Progress was made in understanding the factors needed for an efficient transfection of muscle. An understanding of protein structure and function in muscular dystrophies has allowed elegant examples of protein engineering as a way of gene therapy. Non-viral vectors for gene transfer, targeted gene modification and transcription modulation have also been explored recently. Cell therapy (myogenic-cell transplantation) progressed in understanding myoblast transplantation in primates for human applications, evaluating protocols for the control of graft rejection, understanding the biology of donor myogenic cells, and searching for alternative sources of donor cells. Three clinical trials using pharmacological approaches (anabolic agents and gentamicin) show very poor or negative results. Other pharmacological approaches (upregulation of alternative therapeutic proteins) are still being researched in mice. SUMMARY:This panoply of experimental approaches covered all the current possibilities of attacking the problem of treating muscular dystrophies. It is expected that one or more will progress to provide efficient tools for the ultimate clinical goal: to prolong function and life in severe muscular dystrophy patients.
journal_name
Curr Opin Neuroljournal_title
Current opinion in neurologyauthors
Skuk D,Vilquin JT,Tremblay JPdoi
10.1097/00019052-200210000-00007subject
Has Abstractpub_date
2002-10-01 00:00:00pages
563-9issue
5eissn
1350-7540issn
1473-6551journal_volume
15pub_type
杂志文章,评审abstract::With respect to diverse clinical applications for muscle regeneration, this paper discusses the latest markers for identifying skeletal muscle precursor cells in regenerating muscle, the implications of alternative sources of myogenic precursor cells and putative stem cells, and the current status of administration of...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-199910000-00007
更新日期:1999-10-01 00:00:00
abstract:PURPOSE OF REVIEW:Central nervous system infections are not uncommon in systemic vasculitides. Being vigilant of central nervous system infections as an alternative consideration, instead of systemic vasculitides per se, could prove to be lifesaving in certain circumstances. RECENT FINDINGS:Common pathogens, such as t...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e3283013983
更新日期:2008-06-01 00:00:00
abstract:PURPOSE OF REVIEW:This review focuses on important new findings in the field of tremor and illustrates the consequences for the current definition and classification of tremor. RECENT FINDINGS:Since 1998 when the consensus criteria for tremor were proposed, new variants of tremors and new diagnostic methods were disco...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000211
更新日期:2015-08-01 00:00:00
abstract:PURPOSE OF REVIEW:Hyperglycemia is frequent in patients with cerebrovascular disease. This review article aims to summarize the recent evidence from observational studies that examined the adverse cerebrovascular effects of dysglycemic states as well as interventional studies assessing intensive management strategies f...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e3283418fed
更新日期:2011-02-01 00:00:00
abstract:PURPOSE OF REVIEW:Migraine has traditionally been categorized as a pain disorder, focusing on headache as its central feature. This narrow view does not account for the complex array of premonitory and postrdromal symptoms that occur in the hours before and after headache. This review outlines evidence that supports a ...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e32836085f4
更新日期:2013-06-01 00:00:00
abstract:PURPOSE OF REVIEW:Functional neuro-imaging studies of aphasic stroke offer the potential for a better understanding of the neuronal mechanisms that sustain language recovery. Conclusions, however, have been hampered by a set of unexpected challenges related to experimental design and interpretation. In this review of s...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/01.wco.0000168081.76859.c1
更新日期:2005-08-01 00:00:00
abstract:PURPOSE OF REVIEW:Health-related quality of life is a new outcome variable in neurology. Several generic measures aim at assessing this variable in adults with neurological diseases. Disease-specific measures are still rare; however, individuals with neurological diseases frequently suffer from cognitive impairment, ye...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/01.wco.0000194140.56429.75
更新日期:2005-12-01 00:00:00
abstract:PURPOSE OF REVIEW:The successful motor rehabilitation of stroke, traumatic brain-injured and spinal cord-injured patients requires an intensive and task-specific therapy approach. Budget constraints limit a hand-to-hand therapy approach, so that intelligent machines may offer a solution to promote motor recovery and ob...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/01.wco.0000102630.16692.38
更新日期:2003-12-01 00:00:00
abstract::The motoneuron has a strong ability to regenerate after injury. However, the problems of nerve cell survival after a proximal axotomy, difficulty in axonal elongation after intraspinal lesion and the lack of target specificity during nerve fibre regrowth interfere with a good functional restitution. Current research h...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-200012000-00012
更新日期:2000-12-01 00:00:00
abstract:PURPOSE OF REVIEW:This review focuses on recent advances in the diagnostic approaches and the underlying pathophysiological mechanisms of Charcot-Marie-Tooth (CMT) disease. We also discuss the emerging therapies for this hereditary neuropathy. RECENT FINDINGS:To date, numerous genes are implicated in CMT, and new gene...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e328364c04b
更新日期:2013-10-01 00:00:00
abstract:PURPOSE OF REVIEW:This review aims to describe new features on the epidemiology of encephalitis world-wide. As this neurological presentation is most frequently related to transmitted viruses, surveillance of encephalitis is of major importance to detect their emergence or re-emergence. RECENT FINDINGS:Rabies causes o...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000097
更新日期:2014-06-01 00:00:00
abstract:PURPOSE OF REVIEW:To describe the most important issues a clinician must consider whenever selecting and administering antiepileptic drugs (AEDs). There is no available algorithm that identifies how to individualize selection of AEDs. Proper selection and administration can make an enormous difference in both effective...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000532
更新日期:2018-04-01 00:00:00
abstract::There is a spectrum of acute autonomic neuropathies. Acute panautonomic neuropathy (pandysautonomia), characterized by severe widespread sympathetic and parasympathetic failure, is at one extreme. Guillain-Barré syndrome is at the other end of the spectrum, where the brunt of the disorder falls on the somatic nervous ...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-199410000-00007
更新日期:1994-10-01 00:00:00
abstract::In multifocal motor or sensorimotor neuropathy with persistent conduction block, most of the recent research has concerned treatment. Open trials show that intravenous immunoglobulin is effective, providing a rapid and often marked clinical improvement, accompanied by a significant reduction of conduction block in som...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-199510000-00006
更新日期:1995-10-01 00:00:00
abstract:PURPOSE OF REVIEW:Precise localization of the epileptogenic zone is imperative for the success of resective surgery of drug-resistant epileptic patients. To decrease the number of surgical failures, clinical research has been focusing on finding new biomarkers. For the past decades, high-frequency oscillations (HFOs, 8...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000663
更新日期:2019-04-01 00:00:00
abstract:PURPOSE OF REVIEW:The aim of this article is to review the current literature on endovascular treatment of acute ischemic stroke in the aftermath of the coronavirus disease 2019 (COVID-19) lockdown. RECENT FINDINGS:The outbreak of the COVID-19 has had effect of unprecedented magnitude on the social, economic and perso...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000890
更新日期:2021-02-01 00:00:00
abstract:PURPOSE OF REVIEW:The exotic and emerging viral encephalitides are caused by animal or human viruses and characterised by sudden unexpected outbreaks of neurological disease, usually in tropical and sub-tropical regions, but sometimes spreading to temperate areas. Although a wide range of viruses come within this label...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/01.wco.0000073944.19076.56
更新日期:2003-06-01 00:00:00
abstract:PURPOSE OF REVIEW:The aim is to review recent publications on amyotrophic lateral sclerosis (ALS). RECENT FINDINGS:The Awaji recommendations for electrophysiological diagnosis will permit earlier clinical trials entry. The use of ultrasound to visualize fasciculations, even in deep muscles, will contribute to earlier ...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e32834916a9
更新日期:2011-10-01 00:00:00
abstract:PURPOSE OF REVIEW:The computational power of the brain arises from the complex interactions between neurons. One straightforward method to quantify the strength of neuronal interactions is by measuring correlation and coherence. Efforts to measure correlation have been advancing rapidly of late, spurred by the developm...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000512
更新日期:2018-02-01 00:00:00
abstract:PURPOSE OF REVIEW:The search for a cure for Duchenne muscular dystrophy and other muscular dystrophies is progressing rapidly despite significant challenges posed by some genes and the complexity of targeting all skeletal muscles. This review focuses on three areas in which experimental clinical studies are in progress...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e3282efc157
更新日期:2007-10-01 00:00:00
abstract:PURPOSE OF REVIEW:The last two decades have witnessed a growing concern over the treatment of epilepsy in women of childbearing age, with an increased risk of major congenital malformations and possible cognitive difficulties associated with certain antiepileptic drugs. The aim here is to review the literature regardin...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e3283292401
更新日期:2009-04-01 00:00:00
abstract:PURPOSE OF REVIEW:With the aging of the population, treatment of painful neuropathies is becoming more and more important for neurological practice. This short review highlights recent findings and current problems. RECENT FINDINGS:In addition to tricyclic antidepressants and gabapentin, the reliability of which is es...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e328285dfd6
更新日期:2007-10-01 00:00:00
abstract:PURPOSE OF REVIEW:We analyze recent data on technical aspects, clinical indications, and imaging features of spinal cord MRI in multiple sclerosis, and on the value of this examination for assessing the type and extension of spinal cord damage, and for predicting prognosis in patients with this disease. RECENT FINDING...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000343
更新日期:2016-08-01 00:00:00
abstract::The pathogenesis of primary vasculitides, which are assumed to have an autoimmune pathogenesis, is not well understood. The endothelial cell adhesion molecules seem to play an active role, which varies according to the histopathologic stage of vascular lesions. The role of genetic factors also seems quite important, a...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-199910000-00017
更新日期:1999-10-01 00:00:00
abstract::Trauma to the developing brain constitutes an unexplored field. The few studies attempting to model and study paediatric head trauma, the leading cause of death and disability in the paediatric population, have revealed interesting aspects and potential targets for future research. One feature unique to the developing...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-200004000-00005
更新日期:2000-04-01 00:00:00
abstract:PURPOSE OF REVIEW:This review provides an overview of recent developments in diagnosis, pathophysiology, neuroimaging and management of functional (psychogenic) movement disorders (FMD). RECENT FINDINGS:There has been increasing interest to study the underlying pathophysiology of FMD, which has resulted in a broadened...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e3283551bc1
更新日期:2012-08-01 00:00:00
abstract:PURPOSE OF REVIEW:This review highlights recent work using advanced imaging approaches that have improved our understanding of the underlying neural mechanisms associated with disrupted brain development or demonstrated the potential of MRI to provide objective biomarkers of cerebral injury that relate to subsequent ne...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000073
更新日期:2014-04-01 00:00:00
abstract::Positron emission tomography, single photon emission computed tomography and magnetic resonance spectroscopy provide distinct, uncorrelated and sensitive indicators of brain tumors which can be used to improve differential diagnosis, biopsy guidance or therapy monitoring. New findings, including conflicting ethyl-cyst...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/00019052-199612000-00006
更新日期:1996-12-01 00:00:00
abstract:PURPOSE OF REVIEW:A stroke mimic is a situation in which a diagnosis of stroke at admission is not confirmed, and a stroke chameleon is a situation in which a stroke is revealed by clinical symptoms that are not usual in stroke. The objective of this review is to identify the most frequent clinical situations in which ...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0000000000000620
更新日期:2019-02-01 00:00:00
abstract:PURPOSE OF REVIEW:Recent progress in serological screening for paraneoplastic autoantibodies and diagnostic imaging techniques to detect malignancies has resulted in a broadening of the concept of paraneoplastic neurologic syndromes through the characterization of nonclassical clinical features. The goal of this articl...
journal_title:Current opinion in neurology
pub_type: 杂志文章,评审
doi:10.1097/WCO.0b013e32834a87b7
更新日期:2011-10-01 00:00:00