AAV-based in vivo gene therapy for neurological disorders

Q Ling, JA Herstine, A Bradbury, SJ Gray - Nature Reviews Drug …, 2023 - nature.com
Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

DNA methylation and regulation of gene expression: Guardian of our health

GA Dhar, S Saha, P Mitra, R Nag Chaudhuri - The Nucleus, 2021 - Springer
One of the most critical epigenetic signatures present in the genome of higher eukaryotes is
the methylation of DNA at the C-5 position of the cytosine ring. Based on the sites of DNA …

Phosphoregulated FMRP phase separation models activity-dependent translation through bidirectional control of mRNA granule formation

B Tsang, J Arsenault, RM Vernon… - Proceedings of the …, 2019 - National Acad Sciences
Activity-dependent translation requires the transport of mRNAs within membraneless protein
assemblies known as neuronal granules from the cell body toward synaptic regions …

Developmental studies in fragile X syndrome

KA Razak, KC Dominick, CA Erickson - Journal of neurodevelopmental …, 2020 - Springer
Fragile X syndrome (FXS) is the most common single gene cause of autism and intellectual
disabilities. Humans with FXS exhibit increased anxiety, sensory hypersensitivity, seizures …

Mammalian susceptibility to a neonicotinoid insecticide after fetal and early postnatal exposure

AP Burke, Y Niibori, H Terayama, M Ito, C Pidgeon… - Scientific reports, 2018 - nature.com
Neonicotinoids have become the most widely used class of insecticides world-wide.
Although numerous studies have documented neonicotinoid toxicity in bees and other …

Association between IQ and FMR1 protein (FMRP) across the spectrum of CGG repeat expansions

K Kim, D Hessl, JL Randol, GM Espinal, A Schneider… - PLoS …, 2019 - journals.plos.org
Fragile X syndrome, the leading heritable form of intellectual disability, is caused by
hypermethylation and transcriptional silencing of large (CGG) repeat expansions (> 200 …

Inhibition of glycogen synthase kinase 3 by lithium, a mechanism in search of specificity

D Chatterjee, JM Beaulieu - Frontiers in Molecular Neuroscience, 2022 - frontiersin.org
Inhibition of Glycogen synthase kinase 3 (GSK3) is a popular explanation for the effects of
lithium ions on mood regulation in bipolar disorder and other mental illnesses, including …

What we can learn from a genetic rodent model about autism

D Möhrle, M Fernández, O Peñagarikano… - Neuroscience & …, 2020 - Elsevier
Autism spectrum disorders (ASD) are complex neurodevelopmental disorders that are
caused by genetic and/or environmental impacts, often probably by the interaction of both …

Mechanisms underlying auditory processing deficits in Fragile X syndrome

EA McCullagh, SE Rotschafer, BD Auerbach… - The FASEB …, 2020 - Wiley Online Library
Autism spectrum disorders (ASD) are strongly associated with auditory hypersensitivity or
hyperacusis (difficulty tolerating sounds). Fragile X syndrome (FXS), the most common …

Identification of a molecular locus for normalizing dysregulated GABA release from interneurons in the Fragile X brain

YM Yang, J Arsenault, A Bah, M Krzeminski… - Molecular …, 2020 - nature.com
Principal neurons encode information by varying their firing rate and patterns precisely fine-
tuned through GABAergic interneurons. Dysregulation of inhibition can lead to …