Gene therapy conversion of striatal astrocytes into GABAergic neurons in mouse models of Huntington's disease

Z Wu, M Parry, XY Hou, MH Liu, H Wang, R Cain… - Nature …, 2020 - nature.com
Huntington's disease (HD) is caused by Huntingtin (Htt) gene mutation resulting in the loss
of striatal GABAergic neurons and motor functional deficits. We report here an in vivo cell …

Synaptopathic mechanisms of neurodegeneration and dementia: Insights from Huntington's disease

S Tyebji, AJ Hannan - Progress in Neurobiology, 2017 - Elsevier
Dementia encapsulates a set of symptoms that include loss of mental abilities such as
memory, problem solving or language, and reduces a person's ability to perform daily …

Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

HFE Gleitz, AY Liao, JR Cook, SF Rowlston… - EMBO molecular …, 2018 - embopress.org
The pediatric lysosomal storage disorder mucopolysaccharidosis type II is caused by
mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe …

Test–retest measurements of dopamine D1-type receptors using simultaneous PET/MRI imaging

S Kaller, M Rullmann, M Patt, GA Becker… - European journal of …, 2017 - Springer
Purpose The role of dopamine D 1-type receptor (D 1 R)-expressing neurons in the
regulation of motivated behavior and reward prediction has not yet been fully established …

Pathomechanisms of behavioral abnormalities in Huntington disease: an update

KA Jellinger - Journal of Neural Transmission, 2024 - Springer
Huntington disease (HD), a devastating autosomal-dominant neurodegenerative disease
caused by an expanded CAG trinucleotide repeat, is clinically characterized by a triad of …

The dual role of dopamine in the modulation of information processing in the prefrontal cortex underlying social behavior

H Sotoyama, H Inaba, Y Iwakura, H Namba… - The FASEB …, 2022 - Wiley Online Library
Dopamine in the prefrontal cortex is essential for the regulation of social behavior. However,
stress‐causing social withdrawal also promotes dopamine release in the prefrontal cortex …

Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II

HFE Gleitz, C O'Leary, RJ Holley, BW Bigger - PLoS One, 2017 - journals.plos.org
Severe mucopolysaccharidosis type II (MPS II) is a progressive lysosomal storage disease
caused by mutations in the IDS gene, leading to a deficiency in the iduronate-2-sulfatase …

[HTML][HTML] Phenotypic characterisation of the Mucopolysaccharidosis Type I (MPSI) Idua-W392X mouse model reveals increased anxiety-related traits in female mice

T Andreou, Y Ishikawa-Learmonth… - Molecular Genetics and …, 2023 - Elsevier
Abstract Mucopolysaccharidosis Type I (MPSI) is a rare inherited lysosomal storage disease
that arises due to mutations in the IDUA gene. Defective alpha-L-iduronidase (IDUA) …

Modelling the neuromotor abnormalities of psychotic illness: putative mechanisms and systems dysfunction

JL Waddington, CM O'Tuathaigh - Schizophrenia Research, 2018 - Elsevier
Limitations in access to antipsychotic-naïve patients and in the incisiveness of studies that
can be conducted on them, together with the inevitability of subsequent antipsychotic …

Analysis of gait in rats with olivocerebellar lesions and ability of the nicotinic acetylcholine receptor agonist varenicline to attenuate impairments

CS Lambert, RM Philpot, ME Engberg, BE Johns… - Behavioural brain …, 2015 - Elsevier
Studies have demonstrated that administration of the neuronal nicotinic receptor agonist
varenicline to rats with olivocerebellar lesions attenuates balance deficits on a rotorod and …