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M

Martine Barkats

sorbonne university and inserm

H指数:27

被引数:3,084

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

肌肉干细胞中Smn的靶向敲降诱导了运动神经元非自主性丢失

IF11.7

2026-02-060

OA

Correction to: Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology (vol 132, 257, 2016)

ACTA NEUROPATHOLOGICA

IF9.3

2023-08-040

OA

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

IF5

2021-12-101

OAAI

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

MOLECULAR THERAPY

IF12

2020-08-0129

OAAI

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

TRANSLATIONAL NEURODEGENERATION

IF15.2

2019-12-0528

OAAI

Functional evaluation of an AAV9 based vector expressing alpha-galactosidase A for potential gene therapy of Fabry disease

MOLECULAR GENETICS AND METABOLISM

IF3.5

2019-02-011

PREAI

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

IF9

2018-03-0145

OAAI

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

HUMAN MOLECULAR GENETICS

IF3.2

2018-01-0812

OAAI

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

SCIENCE TRANSLATIONAL MEDICINE

IF14.6

2017-11-29104

OAAI

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

MOLECULAR THERAPY

IF12

2017-09-0160

OAAI

A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome

NEUROBIOLOGY OF DISEASE

IF5.6

2017-03-0139

OAAI

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

NUCLEIC ACIDS RESEARCH

IF13.1

2017-01-2316

OAAI

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

IF17.5

2016-07-2811

OAAI

Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology

ACTA NEUROPATHOLOGICA

IF9.3

2016-04-30160

OAAI

Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases

雌激素介导的AIRE下调影响自身免疫性疾病的性二态性

JOURNAL OF CLINICAL INVESTIGATION

IF13.6

2016-03-21156

OA

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

FRONTIERS IN MOLECULAR NEUROSCIENCE

IF3.8

2015-07-2892

OAAI

The autophagy/lysosome pathway is impaired in SCA7 patients and SCA7 knock-in mice

ACTA NEUROPATHOLOGICA

IF9.3

2014-05-2452

PRE

Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice

IF11.7

2013-03-2160

OA

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice

MOLECULAR THERAPY

IF12

2013-02-01127

OA

A rare recessive distal hereditary motor neuropathy with HSJ1 chaperone mutation

ANNALS OF NEUROLOGY

IF7.7

2012-04-2069

PREAI