The use of nanoparticles for gene therapy in the nervous system.
Nanoparticles represent an alternative to viral vectors for genetic material transfer to the nervous system. However, to increase transfection efficiency in the central nervous system and to decrease toxicity, the design of nanoparticles needs to be improved to enhance blood-brain barrier crossing and endosomal escape. This paper reviews the strategies used to solve these difficulties and covers the use of various nanoparticles including natural inorganic particles, natural polymers, cationic lipids, polyethylenimine derivatives, dendrimers, and carbon-based nanoparticles. The effectiveness, both in vivo and in vitro, of each method to deliver genetic material to neural tissue is discussed.
Unidad Asociada Neurodeath, Departamento de Ciencias Médicas, CSIC-Universidad de Castilla-La Mancha, Albacete, Spain.
Francisco C
FC
Pérez-Martínez
Blanca
B
Carrión
Valentín
V
Ceña
D000818:Animals; D001812:Blood-Brain Barrier / Q000187:drug effects / Q000378:metabolism; D002490:Central Nervous System / Q000187:drug effects / Q000378:metabolism; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D053758:Nanoparticles / Q000008:administration & dosage* / Q000627:therapeutic use*; D009422:Nervous System Diseases / Q000188:drug therapy* / Q000235:genetics* / Q000378:metabolism