Gene therapy for neurological disorders: progress and prospects

BE Deverman, BM Ravina, KS Bankiewicz… - Nature Reviews Drug …, 2018 - nature.com
Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

[HTML][HTML] Gene therapy for neurodegenerative disorders: advances, insights and prospects

W Chen, Y Hu, D Ju - Acta Pharmaceutica Sinica B, 2020 - Elsevier
Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of
neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) …

Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters

B Nieuwenhuis, E Laperrousaz, JR Tribble… - Gene therapy, 2023 - nature.com
Recombinant adeno-associated viral vectors (AAVs) are an effective system for gene
transfer. AAV serotype 2 (AAV2) is commonly used to deliver transgenes to retinal ganglion …

[HTML][HTML] Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex

A Watakabe, M Ohtsuka, M Kinoshita, M Takaji… - Neuroscience …, 2015 - Elsevier
Here we investigated the transduction characteristics of adeno-associated viral vector (AAV)
serotypes 1, 2, 5, 8 and 9 in the marmoset cerebral cortex. Using three constructs that each …

Targeted ablation, silencing, and activation establish glycinergic dorsal horn neurons as key components of a spinal gate for pain and itch

E Foster, H Wildner, L Tudeau, S Haueter… - Neuron, 2015 - cell.com
The gate control theory of pain proposes that inhibitory neurons of the spinal dorsal horn
exert critical control over the relay of nociceptive signals to higher brain areas. Here we …

Controlling AAV tropism in the nervous system with natural and engineered capsids

MJ Castle, HT Turunen, LH Vandenberghe… - Gene Therapy for …, 2016 - Springer
More than one hundred naturally occurring variants of adeno-associated virus (AAV) have
been identified, and this library has been further expanded by an array of techniques for …

Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters

B Nieuwenhuis, B Haenzi, S Hilton… - Gene therapy, 2021 - nature.com
Adeno-associated viral vectors are widely used as vehicles for gene transfer to the nervous
system. The promoter and viral vector serotype are two key factors that determine the …

AAV targeting of glial cell types in the central and peripheral nervous system and relevance to human gene therapy

SJ O'Carroll, WH Cook, D Young - Frontiers in Molecular …, 2021 - frontiersin.org
Different glial cell types are found throughout the central (CNS) and peripheral nervous
system (PNS), where they have important functions. These cell types are also involved in …

Pten deletion promotes regrowth of corticospinal tract axons 1 year after spinal cord injury

K Du, S Zheng, Q Zhang, S Li, X Gao, J Wang… - Journal of …, 2015 - jneurosci.org
Chronic spinal cord injury (SCI) is a formidable hurdle that prevents a large number of
injured axons from crossing the lesion, particularly the corticospinal tract (CST). This study …

AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy

A Kagiava, C Karaiskos, J Richter, C Tryfonos… - Gene therapy, 2021 - nature.com
Mutations in the GJB1 gene, encoding the gap junction (GJ) protein connexin32 (Cx32),
cause X-linked Charcot-Marie-Tooth disease (CMT1X), an inherited demyelinating …