Genetically modified large animal models for investigating neurodegenerative diseases

Co-editing PINK1 and DJ-1 Genes Via Adeno-Associated Virus-Delivered CRISPR/Cas9 System in Adult Monkey Brain Elicits Classical Parkinsonian Phenotype

(2021) Hao Li et al.   Neuroscience Bulletin

PINK1 kinase dysfunction triggers neurodegeneration in the primate brain without impacting mitochondrial homeostasis

(2021) Weili Yang et al.   Protein & Cell

Generation of Transgenic Cynomolgus Monkeys Overexpressing the Gene for Amyloid-β Precursor Protein

(2020) Yasunari Seita et al.   JOURNAL OF ALZHEIMERS DISEASE

Truncation of mutant huntingtin in knock-in mice demonstrates exon1 huntingtin is a key pathogenic form

(2020) Huiming Yang et al.   Nature Communications

The physiological roles of tau and Aβ: implications for Alzheimer’s disease pathology and therapeutics

(2020) Sarah A. Kent et al.   ACTA NEUROPATHOLOGICA

Generation of a Hutchinson–Gilford progeria syndrome monkey model by base editing

(2020) Fang Wang et al.   Protein & Cell

Genome editing in large animals: Current status and future prospects

(2019) Jianguo Zhao et al.   National Science Review

CRISPR/Cas9-mediated PINK1 deletion leads to neurodegeneration in rhesus monkeys

(2019) Weili Yang et al.   CELL RESEARCH

Caspase-4 mediates cytoplasmic accumulation of TDP-43 in the primate brains

(2019) Peng Yin et al.   ACTA NEUROPATHOLOGICA

Efficient base editing for multiple genes and loci in pigs using base editors

(2019) Jingke Xie et al.   Nature Communications

The genetics of Parkinson disease

(2018) Hao Deng et al.   AGEING RESEARCH REVIEWS

Cloning of Macaque Monkeys by Somatic Cell Nuclear Transfer

(2018) Zhen Liu et al.   CELL

A Huntingtin Knockin Pig Model Recapitulates Features of Selective Neurodegeneration in Huntington’s Disease

(2018) Sen Yan et al.   CELL

Use of gene-editing technology to introduce targeted modifications in pigs

(2018) Junghyun Ryu et al.   Journal of Animal Science and Biotechnology

Alzheimer's Disease and Methanol Toxicity (Part 2): Lessons from Four Rhesus Macaques (Macaca mulatta) Chronically Fed Methanol

(2018) Meifeng Yang et al.   JOURNAL OF ALZHEIMERS DISEASE

SIRT6 deficiency results in developmental retardation in cynomolgus monkeys

(2018) Weiqi Zhang et al.   NATURE

Protein misfolding, aggregation, and conformational strains in neurodegenerative diseases

(2018) Claudio Soto et al.   NATURE NEUROSCIENCE

Rodent models for Alzheimer disease

(2018) Jürgen Götz et al.   NATURE REVIEWS NEUROSCIENCE

Mosaicism in CRISPR/Cas9-mediated genome editing

(2018) Maryam Mehravar et al.   DEVELOPMENTAL BIOLOGY

Bridging the gap: large animal models in neurodegenerative research

(2017) S. L. Eaton et al.   MAMMALIAN GENOME

Practical considerations for choosing a mouse model of Alzheimer’s disease

(2017) Joanna L. Jankowsky et al.   Molecular Neurodegeneration

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Neurodegenerative Disease Transmission and Transgenesis in Mice

(2017) Brittany N. Dugger et al.   Cold Spring Harbor Perspectives in Biology

Pathology of Neurodegenerative Diseases

(2017) Brittany N. Dugger et al.   Cold Spring Harbor Perspectives in Biology

APP, PSEN1, and PSEN2 mutations in early-onset Alzheimer disease: A genetic screening study of familial and sporadic cases

(2017) Hélène-Marie Lanoiselée et al.   PLOS MEDICINE

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

AAV1/2-induced overexpression of A53T-α-synuclein in the substantia nigra results in degeneration of the nigrostriatal system with Lewy-like pathology and motor impairment: a new mouse model for Parkinson’s disease

(2017) Chi Wang Ip et al.   Acta Neuropathologica Communications

Behavioral testing of minipigs transgenic for the Huntington gene—A three-year observational study

(2017) Verena Schuldenzucker et al.   PLoS One

Promoting Cas9 degradation reduces mosaic mutations in non-human primate embryos

(2017) Zhuchi Tu et al.   Scientific Reports

2D and 3D Stem Cell Models of Primate Cortical Development Identify Species-Specific Differences in Progenitor Behavior Contributing to Brain Size

(2016) Tomoki Otani et al.   Cell Stem Cell

Common Molecular Pathways in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

(2016) Jochen H. Weishaupt et al.   TRENDS IN MOLECULAR MEDICINE

One-step generation of triple gene-targeted pigs using CRISPR/Cas9 system

(2016) Xianlong Wang et al.   Scientific Reports

Clinical Spectrum of Amyotrophic Lateral Sclerosis (ALS)

(2016) Leslie I. Grad et al.   Cold Spring Harbor Perspectives in Medicine

Mutant Alpha-Synuclein Causes Age-Dependent Neuropathology in Monkey Brain

(2015) W. Yang et al.   JOURNAL OF NEUROSCIENCE

Cytoplasmic mislocalization of RNA splicing factors and aberrant neuronal gene splicing in TDP-43 transgenic pig brain

(2015) Guohao Wang et al.   Molecular Neurodegeneration

Huntington disease

(2015) Gillian P. Bates et al.   Nature Reviews Disease Primers

Wild type human TDP-43 potentiates ALS-linked mutant TDP-43 driven progressive motor and cortical neuron degeneration with pathological features of ALS

(2015) Jacqueline C Mitchell et al.   Acta Neuropathologica Communications

Early Parkinson's disease symptoms in  -synuclein transgenic monkeys

(2015) Y. Niu et al.   HUMAN MOLECULAR GENETICS

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos

(2014) Yuyu Niu et al.   CELL

Germline acquisition of Cas9/RNA-mediated gene modifications in monkeys

(2014) Yongchang Chen et al.   CELL RESEARCH

Species-dependent neuropathology in transgenic SOD1 pigs

(2014) Huaqiang Yang et al.   CELL RESEARCH

Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer

(2014) Xiaoqing Zhou et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

TDP-43 causes differential pathology in neuronal versus glial cells in the mouse brain

(2014) S. Yan et al.   HUMAN MOLECULAR GENETICS

Advancing research diagnostic criteria for Alzheimer's disease: the IWG-2 criteria

(2014) Bruno Dubois et al.   LANCET NEUROLOGY

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Efficient, Complete Deletion of Synaptic Proteins using CRISPR

(2014) Salvatore Incontro et al.   NEURON

A canine model of human aging and Alzheimer's disease

(2013) Elizabeth Head   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Controversies and priorities in amyotrophic lateral sclerosis

(2013) Martin R Turner et al.   LANCET NEUROLOGY

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

A Guide to Neurotoxic Animal Models of Parkinson's Disease

(2013) K. Tieu   Cold Spring Harbor Perspectives in Medicine

Neuropathological Alterations in Alzheimer Disease

(2013) A. Serrano-Pozo et al.   Cold Spring Harbor Perspectives in Medicine

Non-human primate model of amyotrophic lateral sclerosis with cytoplasmic mislocalization of TDP-43

(2012) Azusa Uchida et al.   BRAIN

Nonhuman Primate Models of Alzheimer-Like Cerebral Proteopathy

(2012) Eric Heuer et al.   CURRENT PHARMACEUTICAL DESIGN

Object recognition as a measure of memory in 1–2 years old transgenic minipigs carrying the APPsw mutation for Alzheimer’s disease

(2012) Lene Vammen Søndergaard et al.   TRANSGENIC RESEARCH

Generation of minipigs with targeted transgene insertion by recombinase-mediated cassette exchange (RMCE) and somatic cell nuclear transfer (SCNT)

(2012) Jannik Ejnar Jakobsen et al.   TRANSGENIC RESEARCH

Glial nuclear aggregates of superoxide dismutase-1 are regularly present in patients with amyotrophic lateral sclerosis

(2011) Karin Forsberg et al.   ACTA NEUROPATHOLOGICA

Mutant TDP-43 in motor neurons promotes the onset and progression of ALS in rats

(2011) Cao Huang et al.   JOURNAL OF CLINICAL INVESTIGATION

Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43

(2011) Magdalini Polymenidou et al.   NATURE NEUROSCIENCE

¿Existe la enfermedad de Alzheimer en todos los primates? Afección de Alzheimer en primates no humanos y sus implicaciones fisiopatológicas (I)

(2011) A. Toledano et al.   NEUROLOGIA

Huntington's Disease: Can Mice Lead the Way to Treatment?

(2011) Zachary R. Crook et al.   NEURON

Efficient generation of a biallelic knockout in pigs using zinc-finger nucleases

(2011) J. Hauschild et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs

(2010) Dongshan Yang et al.   HUMAN MOLECULAR GENETICS

An ovine transgenic Huntington's disease model

(2010) J. C. Jacobsen et al.   HUMAN MOLECULAR GENETICS

TAR DNA-binding protein 43 in neurodegenerative disease

(2010) Alice S. Chen-Plotkin et al.   Nature Reviews Neurology

Probing the Biology of Alzheimer's Disease in Mice

(2010) Karen H. Ashe et al.   NEURON

Genetic Animal Models of Parkinson's Disease

(2010) Ted M. Dawson et al.   NEURON

Altered distributions of Gemini of coiled bodies and mitochondria in motor neurons of TDP-43 transgenic mice

(2010) X. Shan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TDP-43 transgenic mice develop spastic paralysis and neuronal inclusions characteristic of ALS and frontotemporal lobar degeneration

(2010) Hans Wils et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Rethinking ALS: The FUS about TDP-43

(2009) Clotilde Lagier-Tourenne et al.   CELL

Absence of nigral degeneration in aged parkin/DJ-1/PINK1 triple knockout mice

(2009) Tohru Kitada et al.   JOURNAL OF NEUROCHEMISTRY

TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration

(2009) I. Wegorzewska et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hemizygous minipigs produced by random gene insertion and handmade cloning express the Alzheimer’s disease-causing dominant mutation APPsw

(2009) Peter M. Kragh et al.   TRANSGENIC RESEARCH

Towards a transgenic model of Huntington’s disease in a non-human primate

(2008) Shang-Hsun Yang et al.   NATURE