In recent years, gene therapy has been raising hopes towards viable treatment strategy for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Diseases of the nervous system have devastating effects and are widely distributed among
the population, being especially prevalent in the elderly. These diseases are often caused …

Gene therapy for rare monogenetic neurological disorders is reaching clinics and offering
hope to families affected by these diseases. There is also potential for gene therapy to offer …

Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors,
use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as …

Gene therapy is making a comeback. With its twin promise of targeting disease etiology and
'long-term correction', gene-based therapies (defined here as all forms of genome …

Given improvements in viral vector design, production and efficiency of transduction in the
central nervous system (CNS), as well as increased knowledge of neuropathological …

Current clinical treatments for central nervous system (CNS) diseases, such as Parkinson's
disease and glioblastoma do not halt disease progression and have significant treatment …

Introduction: Many nervous system disorders are minimally responsive to existing treatments
but they are potential candidates for gene therapy, an approach that can correct the genetic …

Rare diseases collectively exact a high toll on society due to their sheer number and overall
prevalence. Their heterogeneity, diversity, and nature pose daunting clinical challenges for …

Abstract Charcot-Marie-Tooth disease (CMT) encompasses numerous genetically
heterogeneous inherited neuropathies, which together are one of the commonest …