Martine Barkats

Inserm — France

Medicine · Health Sciences

27h-index3.0kcitations51works0.52yr avg
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Research Topics

Neurogenetic and Muscular Disorders Research(23), Virus-based gene therapy research(14), RNA modifications and cancer(10), Muscle Physiology and Disorders(6), Nerve injury and regeneration(6)

Publications51 total

Targeted knockdown of <i>Smn</i> in muscle stem cells induces non-cell autonomous loss of motor neurons
Brain·2026Open Access
Targeted-SMN insufficiency in Skeletal Muscle Stem Cells mediates non-cell autonomous loss of motor neurons at long term
2024· 1 citedOpen Access
Correction to: Intraneuronal aggregation of the β‑CTF fragment of APP (C99) induces Aβ‑independent lysosomal-autophagic pathology
Acta Neuropathologica·2023· 2 citedOpen Access
Splicing efficiency of minor introns in a mouse model of SMA predominantly depends on their branchpoint sequence and can involve the contribution of major spliceosome components
RNA·2021· 3 citedOpen Access
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A
Molecular Therapy — Methods & Clinical Development·2020· 25 citedOpen Access
AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
Molecular Therapy·2020· 50 citedOpen Access
AAV9- mediated expression of SMN restricted to neurons does not rescue the spinal muscular atrophy phenotype
HAL (Le Centre pour la Communication Scientifique Directe)·2020
Amyotrophie spinale infantile
médecine/sciences·2020· 23 citedOpen Access
Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models
Translational Neurodegeneration·2019· 43 citedOpen Access
Functional evaluation of an AAV9 based vector expressing alpha-galactosidase A for potential gene therapy of Fabry disease
Molecular Genetics and Metabolism·2019· 1 cited
Role of the SMN complex and the methylosome in selenoprotein mRNP assembly and translation
HAL (Le Centre pour la Communication Scientifique Directe)·2018Open Access
AAV-mediated gene therapy for fALS
HAL (Le Centre pour la Communication Scientifique Directe)·2018
Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice
Human Molecular Genetics·2018· 17 citedOpen Access
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
Science Translational Medicine·2017· 130 citedOpen Access
A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model
Molecular Therapy·2017· 77 citedOpen Access
SMN replacement restricted to the Central Nervous System does not rescue SMA mice.
HAL (Le Centre pour la Communication Scientifique Directe)·2017
β-Amyloid Precursor Protein Intracellular Domain Controls Mitochondrial Function by Modulating Phosphatase and Tensin Homolog–Induced Kinase 1 Transcription in Cells and in Alzheimer Mice Models
Biological Psychiatry·2017· 59 citedOpen Access
SECIS-binding protein 2 interacts with the SMN complex and the methylosome for selenoprotein mRNP assembly and translation
Nucleic Acids Research·2017· 15 citedOpen Access
A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome
Neurobiology of Disease·2016· 43 citedOpen Access
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins
Molecular Neurodegeneration·2016· 15 citedOpen Access
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Frequent Co-authors

Maria Grazia Biferi(18), Thibaut Marais(16), Stéphanie Astord(15), Jacques Mallet(11), Aurore Besse(7), Sandra Duqué(6), Stéphanie Millecamps(6), Philippe Moullier(5), Mathilde Cohen-Tannoudji(5), Thomas Voït(4), Inger Lauritzen(4), Raphaëlle Pardossi‐Piquard(4), Alexandre Bourgeois(4), Frédéric Checler(4), Sandro Alves(4), Martina Marinello(4), Merle Ruberg(4), Giovanni Stévanin(4), Alexis Brice(4), Annie Sittler(4)