Genetically modified large animal models for investigating neurodegenerative diseases

W Yang, X Chen, S Li, XJ Li - Cell & Bioscience, 2021 - Springer
Neurodegenerative diseases represent a large group of neurological disorders including
Alzheimer's disease, amyotrophic lateral sclerosis, Parkinson's disease, and Huntington's …

New pathogenic insights from large animal models of neurodegenerative diseases

P Yin, S Li, XJ Li, W Yang - Protein & Cell, 2022 - academic.oup.com
Animal models are essential for investigating the pathogenesis and developing the
treatment of human diseases. Identification of genetic mutations responsible for …

AAV5-miHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a Huntington's disease minipig model

MM Evers, J Miniarikova, S Juhas, A Vallès… - Molecular Therapy, 2018 - cell.com
Huntington's disease (HD) is a fatal neurodegenerative disorder caused by a CAG
trinucleotide repeat expansion in the huntingtin gene. Previously, we showed strong …

[HTML][HTML] Growth hormone receptor-deficient pigs resemble the pathophysiology of human Laron syndrome and reveal altered activation of signaling cascades in the …

A Hinrichs, B Kessler, M Kurome, A Blutke, E Kemter… - Molecular …, 2018 - Elsevier
Objective Laron syndrome (LS) is a rare, autosomal recessive disorder in humans caused
by loss-of-function mutations of the growth hormone receptor (GHR) gene. To establish a …

The effects of huntingtin-lowering: what do we know so far?

WF Kaemmerer, RC Grondin - Degenerative neurological and …, 2019 - Taylor & Francis
Therapies targeting mutant huntingtin DNA, mRNA, and protein have a chance at becoming
the first disease-modifying treatments for Huntington's disease, a fatal inherited …

Large animals in neurointerventional research: a systematic review on models, techniques and their application in endovascular procedures for stroke, aneurysms …

AM Herrmann, S Meckel, MJ Gounis… - Journal of Cerebral …, 2019 - journals.sagepub.com
Neuroendovascular procedures have led to breakthroughs in the treatment of ischemic
stroke, intracranial aneurysms, and intracranial arteriovenous malformations. Due to these …

Pig models of neurodegenerative disorders: Utilization in cell replacement‐based preclinical safety and efficacy studies

D Dolezalova, M Hruska‐Plochan… - Journal of …, 2014 - Wiley Online Library
An important component for successful translation of cell replacement‐based therapies into
clinical practice is the utilization of large animal models to conduct efficacy and/or safety cell …

[HTML][HTML] Influence of 3-nitropropionic acid on physiological and behavioral responses in zebrafish larvae and adults

MT Wiprich, R Zanandrea, S Altenhofen… - … and Physiology Part C …, 2020 - Elsevier
Long-term treatment with 3-nitropropionic acid (3-NPA), a toxin derived from plants and
fungi, may reproduce symptoms and biochemical characteristics of Huntington's disease …

Large animal models of Huntington's disease: what we have learned and where we need to go next

D Howland, Z Ellederova, N Aronin… - Journal of …, 2020 - content.iospress.com
Genetically modified rodent models of Huntington's disease (HD) have been especially
valuable to our understanding of HD pathology and the mechanisms by which the mutant …

Nervous system sampling for general toxicity and neurotoxicity studies in the laboratory minipig with emphasis on the Göttingen minipig

ID Pardo, RA Manno, R Capobianco… - Toxicologic …, 2021 - journals.sagepub.com
The use of minipigs as an alternative nonclinical species has increased in the last 20 years.
The Society of Toxicologic Pathology (STP) has produced generic “best practice” …