Genome Surgery & Neuronal Disorders

Genome surgery using CRISPR genomic techniques offer the potential to correct disease-causing mutations in many neuronal disorders. Here are the latest discoveries pertaining to genome surgery and neuronal disorders.

February 12, 2021
Preprint
Open Access

Targeting Gys1 with AAV-SaCas9 decreases pathogenic polyglucosan bodies and neuroinflammation in Adult Polyglucosan Body and Lafora disease mouse models

BioRxiv : the Preprint Server for Biology
E. GumusgozBerge A Minassian
February 12, 2021
Preprint
Open Access

Phenotypic analysis of catastrophic childhood epilepsy genes: The Epilepsy Zebrafish Project

BioRxiv : the Preprint Server for Biology
A. GriffinScott C Baraban
February 11, 2021

REST protects dopaminergic neurons from mitochondrial and α-synuclein oligomer pathology in an alpha synuclein overexpressing BAC-transgenic mouse model.

The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
B J RyanR Wade-Martins
January 23, 2021

CRISPR/Cas9-mediated grna gene knockout leads to neurodevelopmental defects and motor behavior changes in zebrafish.

Journal of Neurochemistry
Jiuling ZhuHaibin Xia
January 8, 2021
Open Access

CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice

The Journal of Clinical Investigation
Ralf S SchmidJames M Wilson
December 20, 2020
Review
Open Access

Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases

International Journal of Molecular Sciences
Wei ChiuHao-Min Cheng
December 12, 2020
Review

Gene therapy using haematopoietic stem and progenitor cells

Nature Reviews. Genetics
Giuliana FerrariAlessandro Aiuti
November 19, 2020
Preprint
Open Access

HTT silencing delays onset and slows progression of Huntington disease like phenotype: Monitoring with a novel neurovascular biomarker

BioRxiv : the Preprint Server for Biology
H. LiuWenzhen Duan
November 18, 2020
Open Access

CRISPR-Mediated Induction of Neuron-Enriched Mitochondrial Proteins Boosts Direct Glia-to-Neuron Conversion

Cell Stem Cell
Gianluca L RussoMagdalena Götz
November 17, 2020
Review
Open Access

Genome Editing for CNS Disorders

Frontiers in Neuroscience
Fábio Duarte, Nicole Déglon
November 3, 2020
Open Access

Epilepsy and neurobehavioral abnormalities in mice with a dominant-negative KCNB1 pathogenic variant

Neurobiology of Disease
Nicole A HawkinsJennifer A Kearney
October 27, 2020

Peptide Shuttle-Mediated Delivery for Brain Gene Therapies

Current Topics in Medicinal Chemistry
Josep GarciaMeritxell Teixidó
October 6, 2020
Open Access

Development and Validation of CRISPR Activator Systems for Overexpression of CB1 Receptors in Neurons

Frontiers in Molecular Neuroscience
Valentina Di MariaMarco Ledri
September 29, 2020
Review
Open Access

Collaborative Efforts for Spinocerebellar Ataxia Research in the United States: CRC-SCA and READISCA

Frontiers in Neurology
Chih-Chun LinSheng-Han Kuo
September 27, 2020

Commentary on Some Recent Theses Relevant to Combating Aging: October 2020

Rejuvenation Research
Benjamin Zealley, Aubrey D N J de Grey
September 10, 2020
Review
Open Access

Targeting Alternative Splicing as a Potential Therapy for Episodic Ataxia Type 2

Biomedicines
Fanny JaudonLorenzo A Cingolani
September 10, 2020
Open Access

Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases.

Frontiers in Molecular Neuroscience
Joseph Edward RittinerBoris Kantor
August 1, 2020
Review

CRISPR/Cas9 novel therapeutic road for the treatment of neurodegenerative diseases

Life Sciences
Ansar KarimianBahman Yousefi

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