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RESEARCH PRODUCT
Non-primate lentiviral vectors and their applications in gene therapy for ocular disorders
Elena BaiamonteMelania Lo IaconoVincenzo Cavalierisubject
0301 basic medicineEye DiseasesGenetic enhancementGenetic Vectorslcsh:QR1-502Settore BIO/11 - Biologia MolecolareReviewComputational biologyGenomelcsh:MicrobiologyLentiviruViral vectorEIAV03 medical and health sciences0302 clinical medicineGene therapyVirologyJDVAnimalsHumansZebrafishDrug CarrierZebrafishDrug CarriersBIVbiologyAnimalLentivirusCAEVEye DiseaseGenetic Therapybiology.organism_classificationFIVOphthalmologyDisease Models Animal030104 developmental biologyInfectious DiseasesVMVLentiviral vector030217 neurology & neurosurgeryHumandescription
Lentiviruses have a number of molecular features in common, starting with the ability to integrate their genetic material into the genome of non-dividing infected cells. A peculiar property of non-primate lentiviruses consists in their incapability to infect and induce diseases in humans, thus providing the main rationale for deriving biologically safe lentiviral vectors for gene therapy applications. In this review, we first give an overview of non-primate lentiviruses, highlighting their common and distinctive molecular characteristics together with key concepts in the molecular biology of lentiviruses. We next examine the bioengineering strategies leading to the conversion of lentiviruses into recombinant lentiviral vectors, discussing their potential clinical applications in ophthalmological research. Finally, we highlight the invaluable role of animal organisms, including the emerging zebrafish model, in ocular gene therapy based on non-primate lentiviral vectors and in ophthalmology research and vision science in general.
year | journal | country | edition | language |
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2018-06-09 |