Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
TLDR
The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery.Abstract:
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and efficiency in transducing a wide range of cell types. The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery.read more
Citations
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Journal ArticleDOI
Adeno-associated virus vector as a platform for gene therapy delivery
TL;DR: The fundamentals of AAV and vectorology are discussed, focusing on current therapeutic strategies, clinical progress and ongoing challenges.
Journal ArticleDOI
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
Ken Y. Chan,Min Jee Jang,Bryan B. Yoo,Alon Greenbaum,Namita Ravi,Wei Li Wu,Luis Óscar Sánchez-Guardado,Carlos Lois,Sarkis K. Mazmanian,Benjamin E. Deverman,Viviana Gradinaru +10 more
TL;DR: When used with cell-type-specific promoters and enhancers, these AAVs enable efficient and targetable genetic modification of cells throughout the nervous system of transgenic and non-transgenic animals.
Journal ArticleDOI
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy.
TL;DR: This review will provide an overview of some important factors to consider in the use of AAV as a vector for gene therapy.
Journal ArticleDOI
Not just amyloid: physiological functions of the amyloid precursor protein family
TL;DR: Focus is focused on the in vivo roles of APP family members and their processing products for CNS development, synapse formation and function, brain injury and neuroprotection, as well as ageing.
Journal ArticleDOI
Drug-Resistant Epilepsy: Multiple Hypotheses, Few Answers
TL;DR: Evidence for and against several major theories trying to explain refractory epilepsy are summarized, including the pharmacokinetic hypothesis, neural network hypothesis, intrinsic severity hypothesis, gene variant hypothesis, target hypothesis, and transporter hypothesis.
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