Add to ORKGGene therapy (GT) has tremendous potential for the treatment of neurological disorders to transform patient care. The successful application of virus-mediated GT to treat spinal muscular atrophy is a significant milestone, serving to accelerate similar progress in a spectrum of neurological conditions, with more than 50 clinical trials currently underway, across neurodevelopmental, neurodegenerative, muscular dystrophy, epilepsy, chronic pain and neoplastic diseases. This review provides an overview of the key features of virus-mediated GT, paradigms of delivery and dosing, potential risks and highlights ongoing research to optimise safe and effective delivery of vectors into the nervous system. Examples of the application of GT in various ...
Background: Neurodevelopmental disorders, as a class of diseases, have been particularly difficult t...
Neurodegenerative disorders (NDDs), such as Alzheimer’s disease (AD) and Parkinson’s Disease (PD), a...
Treating monogenic disorders via gene therapy although still considered experimental by some, has be...
Gene therapy (GT) has tremendous potential for the treatment of neurological disorders to transform ...
The recent success of first central nervous system gene therapies has reinvigorated the growing comm...
Gene therapy consists of the introduction of genetic material into cells for a therapeutic purpose. ...
Diseases of the nervous system have devastating effects and are widely distributed among the populat...
The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous syst...
Gene therapy for rare monogenetic neurological disorders is reaching clinics and offering hope to fa...
In recent years, gene therapy has been raising hopes toward viable treatment strategies for rare gen...
Gene therapy is making a comeback. With its twin promise of targeting disease etiology and 'long-ter...
International audienceFor more than ten years, gene therapy for neurological diseases has experience...
The potential benefits of gene therapy for neurological diseases such as Parkinson’s, Amyotrophic La...
Introduction: We sought to provide an overview of the published and currently ongoing movement disor...
Introduction. Vector-based intracerebral gene therapies are being used to treat specific neurodegene...
Background: Neurodevelopmental disorders, as a class of diseases, have been particularly difficult t...
Neurodegenerative disorders (NDDs), such as Alzheimer’s disease (AD) and Parkinson’s Disease (PD), a...
Treating monogenic disorders via gene therapy although still considered experimental by some, has be...
Gene therapy (GT) has tremendous potential for the treatment of neurological disorders to transform ...
The recent success of first central nervous system gene therapies has reinvigorated the growing comm...
Gene therapy consists of the introduction of genetic material into cells for a therapeutic purpose. ...
Diseases of the nervous system have devastating effects and are widely distributed among the populat...
The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous syst...
Gene therapy for rare monogenetic neurological disorders is reaching clinics and offering hope to fa...
In recent years, gene therapy has been raising hopes toward viable treatment strategies for rare gen...
Gene therapy is making a comeback. With its twin promise of targeting disease etiology and 'long-ter...
International audienceFor more than ten years, gene therapy for neurological diseases has experience...
The potential benefits of gene therapy for neurological diseases such as Parkinson’s, Amyotrophic La...
Introduction: We sought to provide an overview of the published and currently ongoing movement disor...
Introduction. Vector-based intracerebral gene therapies are being used to treat specific neurodegene...
Background: Neurodevelopmental disorders, as a class of diseases, have been particularly difficult t...
Neurodegenerative disorders (NDDs), such as Alzheimer’s disease (AD) and Parkinson’s Disease (PD), a...
Treating monogenic disorders via gene therapy although still considered experimental by some, has be...