Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.
Haurigot V, Marcó S, Ribera A, Garcia M, Ruzo A, Villacampa P, Ayuso E, Añor S, Andaluz A, Pineda M, García-Fructuoso G, Molas M, Maggioni L, Muñoz S, Motas S, Ruberte J, Mingozzi F, Pumarola M, Bosch F.
Haurigot V, et al. Among authors: ruzo a.
J Clin Invest. 2013 Aug 1;123(8):3254-3271. doi: 10.1172/JCI66778. Epub 2013 Jul 1.
J Clin Invest. 2013.
PMID: 23863627
Free PMC article.