Regulatory considerations for translating gene therapy: a European Union perspective.
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.
Galli
Maria Cristina
MC
Department of Cell Biology and Neurosciences, Istituto Superiore di Sanità, Viale Regina Elena 299, Rome, Italy. mariacristina.galli@iss.it
D000818:Animals; D005062:European Union; D015316:Genetic Therapy / Q000331:legislation & jurisprudence*; D017408:Guidelines as Topic; D006801:Humans