Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases
Estera Rintz, Takashi Higuchi, Hiroshi Kobayashi, Deni S. Galileo, Grzegorz Węgrzyn, Shunji Tomatsu
Abstract
lentiviral-mediated gene therapy needs optimization in dose, time of delivery, and promoter-driven expression. Choosing suitable promoters seems to be one of the important factors for the effective expression of the dysfunctional enzyme. This review summarizes the research on therapy for LSDs that has used different lentiviral vectors, emphasizing gene promoters.
Topics & Concepts
Viral vectorComputational biologyLysosomal storage disordersVirologyComputer scienceBiologyRecombinant DNABiochemistryEnzymeGeneLysosomal Storage Disorders ResearchVirus-based gene therapy researchCytomegalovirus and herpesvirus research