Lentiviral Vectors for Ocular Gene Therapy

This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. First, the description of the global challenges encountered to develop safe and efficient lentiviral recombinant vectors for clinical applicatio...

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Bibliographic Details
Published in:Pharmaceutics 2022-07, Vol.14 (8), p.1605
Main Authors: Arsenijevic, Yvan, Berger, Adeline, Udry, Florian, Kostic, Corinne
Format: Article
Language:English
Subjects:
Care and treatment
Cell cycle
Control
Diagnosis
Eye diseases
Gene therapy
Genetic aspects
Genetic vectors
Genomes
glaucoma
Glycoproteins
Identification and classification
Immune system
lentivirus
Mutagenesis
ocular delivery
Patient outcomes
Plasmids
Proteins
retinal disease
Review
RNA viruses
Vectors (Biology)
viral vector
Viruses
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Description
Summary:This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. First, the description of the global challenges encountered to develop safe and efficient lentiviral recombinant vectors for clinical application is provided. The risks and the measures taken to minimize secondary effects as well as new strategies using these vectors are also discussed. This review then focuses on lentiviral vectors specifically designed for ocular therapy and goes over preclinical and clinical studies describing their safety and efficacy. A therapeutic approach using lentiviral vector-mediated gene therapy is currently being developed for many ocular diseases, e.g., aged-related macular degeneration, retinopathy of prematurity, inherited retinal dystrophies (Leber congenital amaurosis type 2, Stargardt disease, Usher syndrome), glaucoma, and corneal fibrosis or engraftment rejection. In summary, this review shows how lentiviral vectors offer an interesting alternative for gene therapy in all ocular compartments.
ISSN:1999-4923
1999-4923
DOI:10.3390/pharmaceutics14081605