Viral vectors for therapy of neurologic diseases

SR Choudhury, E Hudry, CA Maguire… - …, 2017 - Elsevier
Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

Early-life inflammation promotes depressive symptoms in adolescence via microglial engulfment of dendritic spines

P Cao, C Chen, A Liu, Q Shan, X Zhu, C Jia, X Peng… - Neuron, 2021 - cell.com
Early-life inflammation increases the risk for depression in later life. Here, we demonstrate
how early-life inflammation causes adolescent depressive-like symptoms: by altering the …

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

JM Levy, WH Yeh, N Pendse, JR Davis… - Nature biomedical …, 2020 - nature.com
The success of base editors for the study and treatment of genetic diseases depends on the
ability to deliver them in vivo to the relevant cell types. Delivery via adeno-associated viruses …

In vivo target gene activation via CRISPR/Cas9-mediated trans-epigenetic modulation

HK Liao, F Hatanaka, T Araoka, P Reddy, MZ Wu, Y Sui… - Cell, 2017 - cell.com
Current genome-editing systems generally rely on inducing DNA double-strand breaks
(DSBs). This may limit their utility in clinical therapies, as unwanted mutations caused by …

High-speed multiplane confocal microscopy for voltage imaging in densely labeled neuronal populations

TD Weber, MV Moya, K Kılıç, J Mertz… - Nature …, 2023 - nature.com
Genetically encoded voltage indicators (GEVIs) hold immense potential for monitoring
neuronal population activity. To date, best-in-class GEVIs rely on one-photon excitation …

An astrocyte BMAL1-BAG3 axis protects against alpha-synuclein and tau pathology

PW Sheehan, CJ Nadarajah, MF Kanan, JN Patterson… - Neuron, 2023 - cell.com
The circadian clock protein BMAL1 modulates glial activation and amyloid-beta deposition
in mice. However, the effects of BMAL1 on other aspects of neurodegenerative pathology …

Ionizable lipid nanoparticles for therapeutic base editing of congenital brain disease

R Palanki, SK Bose, A Dave, BM White, C Berkowitz… - ACS …, 2023 - ACS Publications
Delivery of mRNA-based therapeutics to the perinatal brain holds great potential in treating
congenital brain diseases. However, nonviral delivery platforms that facilitate nucleic acid …

Alpha-synuclein is a DNA binding protein that modulates DNA repair with implications for Lewy body disorders

AJ Schaser, VR Osterberg, SE Dent, TL Stackhouse… - Scientific reports, 2019 - nature.com
Alpha-synuclein is a presynaptic protein that forms abnormal cytoplasmic aggregates in
Lewy body disorders. Although nuclear alpha-synuclein localization has been described, its …

Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA

JM Wolter, H Mao, G Fragola, JM Simon, JL Krantz… - Nature, 2020 - nature.com
Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by a mutation or
deletion of the maternally inherited UBE3A allele. In neurons, the paternally inherited …

C9ORF72 repeat expansions in mice cause TDP-43 pathology, neuronal loss, and behavioral deficits

J Chew, TF Gendron, M Prudencio, H Sasaguri… - Science, 2015 - science.org
The major genetic cause of frontotemporal dementia and amyotrophic lateral sclerosis is a
G4C2 repeat expansion in C9ORF72. Efforts to combat neurodegeneration associated with …