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Marina Roé

Marina Roé

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Optimization of viral vectors for the effective gene delivery to muscle tissue cells 

Optimization of viral vectors for the effective gene delivery to muscle tissue cells 

Proyectos

Directed evolution of adeno-associated virus (AAV) capsids for effective gene delivery to the muscle fibro-adipogenic progenitors (FAPS)  PROJECT DESCRIPTION  In...

0
A new nanoparticle-based delivery system for nucleic acids to treat Collagen VI-CMD 

A new nanoparticle-based delivery system for nucleic acids to treat Collagen VI-CMD 

DYSTRO-SMARTY: a non-liposomal nanovesicle platform for delivery of  nucleic acids to treat Collagen VI-related Congenital Muscular Dystrophy  PROJECT DESCRIPTION  Although...

0
Personalized medicine for Collagen VI Congenital Muscular Dystrophies

Personalized medicine for Collagen VI Congenital Muscular Dystrophies

Proyectos

Project description: Congenital Muscular Dystrophies are a group of rare, highly disabling neuromuscular diseases with a reduced life expectancy. Over...

0
Update of the international patient registry of Congenital Muscular Dystrophies (CMDIR)

Update of the international patient registry of Congenital Muscular Dystrophies (CMDIR)

Proyectos

Project description: The International Registry of Congenital Muscular Dystrophies (CMDIR) was created in 2009 by CureCMD, a patient association of...

0
RNA editing by recruiting endogenous ADAR using long RNAs to correct glycine substitutions in COL6-RD

RNA editing by recruiting endogenous ADAR using long RNAs to correct glycine substitutions in COL6-RD

Proyectos
0
Study of gene therapy by “exon skipping” for intronic mutations in the pre-clinical phase

Study of gene therapy by “exon skipping” for intronic mutations in the pre-clinical phase

Proyectos
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