Adeno-associated virus-based gene therapy for CNS diseases

M Hocquemiller, L Giersch, M Audrain… - Human gene …, 2016 - liebertpub.com
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 …

The endocannabinoid system as a target for novel anxiolytic drugs

S Patel, MN Hill, JF Cheer, CT Wotjak… - … & Biobehavioral Reviews, 2017 - Elsevier
The endocannabinoid (eCB) system has attracted attention for its role in various behavioral
and brain functions, and as a therapeutic target in neuropsychiatric disease states, including …

[HTML][HTML] Fight fire with fire: Neurobiology of capsaicin-induced analgesia for chronic pain

V Arora, JN Campbell, MK Chung - Pharmacology & therapeutics, 2021 - Elsevier
Capsaicin, the pungent ingredient in chili peppers, produces intense burning pain in
humans. Capsaicin selectively activates the transient receptor potential vanilloid 1 (TRPV1) …

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse

DJ Schuster, JA Dykstra, MS Riedl, KF Kitto… - Frontiers in …, 2014 - frontiersin.org
Adeno-associated virus serotype 9 (AAV9)-mediated gene transfer has been reported in
central nervous system (CNS) and peripheral tissues. The current study compared the …

RNA interference (RNAi)-based therapeutics for treatment of rare neurologic diseases

ND Germain, WK Chung, PD Sarmiere - Molecular Aspects of Medicine, 2023 - Elsevier
Advances in genome sequencing have greatly facilitated the identification of genomic
variants underlying rare neurodevelopmental and neurodegenerative disorders …

Neuronal TRPV1-CGRP axis regulates bone defect repair through Hippo signaling pathway

Y Jiang, Z Zhu, B Wang, Y Yuan, Q Zhang, Y Li, Y Du… - Cellular …, 2023 - Elsevier
Transient receptor potential vanilloid type 1 (TRPV1) is highly expressed on sensory
neurons where it serves as a polymodal receptor for detecting physical and chemical stimuli …

Targeted ubiquitination of sensory neuron calcium channels reduces the development of neuropathic pain

L Sun, CK Tong, TJ Morgenstern… - Proceedings of the …, 2022 - National Acad Sciences
Neuropathic pain caused by lesions to somatosensory neurons due to injury or disease is a
widespread public health problem that is inadequately managed by small-molecule …

Injectable, antioxidative, and neurotrophic factor-deliverable hydrogel for peripheral nerve regeneration and neuropathic pain relief

Y Kong, W Shi, D Zhang, X Jiang, M Kuss, B Liu… - Applied Materials …, 2021 - Elsevier
Although peripheral nerve system has intrinsic regeneration potential, recovery from
peripheral nerve injury (PNI) is often far from optimal, leading to partial or complete loss of …

AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

N Hardcastle, NM Boulis, T Federici - Expert opinion on biological …, 2018 - Taylor & Francis
Introduction: Adeno-associated viral (AAV) vector-mediated gene delivery to the spinal cord
has finally entered the pathway towards regulatory approval. Phase 1 clinical trials using …

AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 deficiency

L Zhu, B Tan, SS Dwight, B Beahm, M Wilsey… - … Therapy-Methods & …, 2022 - cell.com
N-glycanase 1 (NGLY1) Deficiency is a progressive, ultra-rare, autosomal recessive
disorder with no approved therapy and five core clinical features: severe global …