Royal Holloway and Bedford New College

Genethon

Centre National De La Recherche Scientifique

Institut National De La Sante Et De La Recherche Medicale

Sorbonne Université

Centre Hospitalier Universitaire De Nantes

Association Institut De Myologie

Nantes Université

George Dickson

Caroline Le Guiner

Philippe Moullier

Thomas Voit

Linda Popplewell

A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.

Efficient systemic treatment of dystrophic muscle pathologies

There is described a nucleic acid molecule comprising a nucleotide sequence encoding a functional dystrophin protein. Also described is a vector, a host cell and a pharmaceutical composition comprising the nucleic acid molecule; use of the nucleic acid molecule in therapy, such as in the treatment of a muscular dystrophy; and a method of treating muscular dystrophy, the method comprising administering a therapeutically effective amount of the nucleic acid molecule to a patient suffering from a muscular dystrophy.

Treatment of muscular dystrophies

A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein:

An adeno-associated viral (AAV) vector containing an expression construct, wherein: the expression construct comprises a nucleic acid sequence which encodes a microdystrophin (MD); and the nucleic acid sequence encoding the MD has a size of at least 4.1 kb.

Production of large-sized microdystrophins in an AAV-based vector configuration

A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: &#x2014;the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; &#x2014;the composition is systemically administered.

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London, United Kingdom

George Dickson