Martine Barkats

Paris, 75013, France

Last Updated: 09/26/2026

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Found 27 publications

Targeted knockdown of Smn in muscle stem cells induces non-cell autonomous loss of motor neurons.

Targeted knockdown of Smn in muscle stem cells induces non-cell autonomous loss of motor neurons.

Journal: Brain : a journal of neurology

Published: January 26, 2025

Correction to: Intraneuronal aggregation of the β‑CTF fragment of APP (C99) induces Aβ‑independent lysosomal-autophagic pathology.

Correction to: Intraneuronal aggregation of the β‑CTF fragment of APP (C99) induces Aβ‑independent lysosomal-autophagic pathology.

Journal: Acta neuropathologica

Published: August 04, 2023

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.

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.

Journal: RNA (New York, N.Y.)

Published: November 09, 2020

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.

Journal: Molecular therapy. Methods & clinical development

Published: July 13, 2020

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: March 12, 2020

SMA: from gene discovery to gene therapy

SMA: from gene discovery to gene therapy

Journal: Medecine sciences : M/S

Published: March 05, 2020

Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models.

Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models.

Journal: Translational neurodegeneration

Published: August 10, 2019

Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice.

Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice.

Journal: Human molecular genetics

Published: November 19, 2017

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Journal: Science translational medicine

Published: December 23, 2016

A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.

A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: December 15, 2016

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.

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.

Journal: Biological psychiatry

Published: August 26, 2016

SECIS-binding protein 2 interacts with the SMN complex and the methylosome for selenoprotein mRNP assembly and translation.

SECIS-binding protein 2 interacts with the SMN complex and the methylosome for selenoprotein mRNP assembly and translation.

Journal: Nucleic acids research

Published: August 24, 2016

Showing 1-12 of 27

Last Updated: 09/26/2026