title: Human gene therapy: novel approaches to improve the current gene delivery systems.
abstract: Even though gene therapy made its way through the clinics to treat a number of human pathologies since the early years of experimental research and despite the recent approval of the first gene-based product (Glybera) in Europe, the safe and effective use of gene transfer vectors remains a challenge in human gene therapy due to the existence of barriers in the host organism. While work is under active investigation to improve the gene transfer systems themselves, the use of controlled release approaches may offer alternative, convenient tools of vector delivery to achieve a performant gene transfer in vivo while overcoming the various physiological barriers that preclude its wide use in patients. This article provides an overview of the most significant contributions showing how the principles of controlled release strategies may be adapted for human gene therapy.
authors.affiliation: Center of Experimental Orthopaedics, Saarland University Medical Center, Kirrbergerstr, Bldg 37, D-66421 Homburg/Saar, Germany.
authors.forename: Magali
authors.initials: M
authors.lastname: Cucchiarini
mesh_terms: D056704:Adaptive Immunity; D001672:Biocompatible Materials / Q000737:chemistry; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques / Q000009:adverse effects / Q000639:trends*; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000008:administration & dosage / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D006801:Humans; D020100:Hydrogels / Q000737:chemistry; D008072:Hyperlipoproteinemia Type I / Q000628:therapy*; D007113:Immunity, Innate; D013812:Therapeutics / Q000379:methods* / Q000639:trends; D023822:Tissue Engineering
chemical_list: D001672:Biocompatible Materials; D020100:Hydrogels