title: Regulatory considerations for translating gene therapy: a European Union perspective.
abstract: A preclinical study on a gene therapy approach for treatment of the severe muscle weakness associated with a variety of neuromuscular disorders provides a forum to discuss the translational challenges of gene therapy from a regulatory point of view. In this Perspective, the findings are considered from the view of European regulatory requirements for first clinical use.
authors.affiliation: Department of Cell Biology and Neurosciences, Istituto Superiore di Sanità, Viale Regina Elena 299, Rome, Italy. mariacristina.galli@iss.it
authors.forename: Maria Cristina
authors.initials: MC
authors.lastname: Galli
mesh_terms: D000818:Animals; D005062:European Union; D015316:Genetic Therapy / Q000331:legislation & jurisprudence*; D017408:Guidelines as Topic; D006801:Humans