Article Addendum www.landesbioscience.com rare diseases e27209-1 rare diseases 1, e27209; 2013; © 2013 landes Bioscience Article Addendum

Mucopolysaccharidosis Type IIIA (MPSIIIA) represents an unmet medical need. MPSIIIA shares with many other lysosomal storage disorders (LSD) the characteristic of being a severe neurodegenerative disease accompanied by mild somatic involvement. Thus, the main target organ for the development of new treatments is the central nervous system (CNS), but overall clinical effi-cacy would be greatly enhanced by simul-taneous correction of peripheral disease. We have recently developed a novel treat-ment for MPSIIIA based on the delivery to the cerebrospinal fluid of serotype 9 adeno-associated virus (AAV9)-derived vectors. This gene therapy strategy cor-rected both CNS and somatic pathology in animal models through widespread transduction of CNS, peripheral nervous system (PNS), and liver. The work set the grounds for the clinical translation of the approach to treat MPSIIIA in humans. Here we discuss some important consid-erations that further support the appli-cability of this treatment to MPSIIIA and other LSD with CNS and somatic involvement