Fanny Collaud

Genethon (France)

France

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Articles (12)

Year:

2024

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2024

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2023

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2023

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2023

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2021

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2021

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2020

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2020

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2020

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2020

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2020

Collaborators (6)

Maria Grazia Biferi

Sorbonne Université

FRANCE

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Peter J. Lenting

Université Paris-Saclay

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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David Gross

INSERM

FRANCE

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Nathalie Daniele

Head of preclinical research platform

FRANCE

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Fanny Collaud

Research Interests

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Articles (12)

Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa<sup>-/-</sup> mice.

Gene Therapy in Patients with the Crigler-Najjar Syndrome.

Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor.

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

Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors.

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors.

Efficacy of AAV8-h<i>UGT1A1</i> with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.

Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.

Collaborators (6)

Maria Grazia Biferi

Peter J. Lenting

Mathilde Cohen-Tannoudji

Elena Barbon

David Gross

Nathalie Daniele

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