Maria Grazia Biferi

Sorbonne Université

France

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Articles (9)

Year:

2022

Year:

2022

Year:

2021

Year:

2021

Year:

2021

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2020

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2020

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2020

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2020

Collaborators (6)

NAIRA NAOUAR

Sorbonne Université

FRANCE

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Fanny Collaud

Genethon (France)

FRANCE

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Mathilde Cohen-Tannoudji

Institut de Myologie

FRANCE

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Elena Barbon

San Raffaele Telethon Institute for Gene Therapy

ITALY

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Nathalie Daniele

Head of preclinical research platform

FRANCE

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Gisèle Bonne

INSERM

FRANCE

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Maria Grazia Biferi

Research Interests

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Contact this professor

Articles (9)

Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice

Biomarkers for C9orf7-ALS in Symptomatic and Pre-symptomatic Patients: State-of-the-art in the New Era of Clinical Trials

The non-muscle ADF/cofilin-1 controls sarcomeric actin filament integrity and force production in striated muscle laminopathies

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

The Potential of Induced Pluripotent Stem Cells to Test Gene Therapy Approaches for Neuromuscular and Motor Neuron Disorders

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

Beyond the Traditional Clinical Trials for Amyotrophic Lateral Sclerosis and The Future Impact of Gene Therapy

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

The identification of novel biomarkers is required to improve adult SMA patient stratification, diagnosis and treatment

Collaborators (6)

NAIRA NAOUAR

Fanny Collaud

Mathilde Cohen-Tannoudji

Elena Barbon

Nathalie Daniele

Gisèle Bonne

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