Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B

• Published in: Gene therapy Vol. 14; no. 7; pp. 584 - 594
• Main Authors: HASHIMOTO, T, GIBBS, D, LILLO, C, AZARIAN, S. M, LESACKI, E, ZHANEG, X.-M, YAN, X.-J, WILLIAMS, D. S
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.04.2007
• Subjects: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Animals; Applied cell therapy and gene therapy; Biological and medical sciences; Biotechnology; Care and treatment; Deafness; Diagnosis; Dyneins - genetics; Fundamental and applied biological sciences. Psychology; Gene Expression; Gene therapy; Genetic Therapy - methods; Genetic Vectors - administration & dosage; Genetic Vectors - genetics; Health aspects; Health. Pharmaceutical industry; Humans; Industrial applications and implications. Economical aspects; Medical sciences; Melanosomes; Mice; Mice, Inbred C57BL; Mice, Knockout; Mice, Mutant Strains; Microscopy, Immunoelectron; Models, Animal; Myosin; Myosin VIIA; Myosins - genetics; Ophthalmology; Phenotypes; Photoreceptor Cells, Vertebrate - metabolism; Physiological aspects; Pigment Epithelium of Eye - metabolism; Retina; Retina - metabolism; Retinal degeneration; Retinal Degeneration - metabolism; Retinal Degeneration - therapy; Retinopathies; Risk factors; Rod Opsins - metabolism; Rodents; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transgenes; Usher Syndromes - therapy; Usher's syndrome; United States; Animal model; Replacement therapy; Retinopathy; RPE; Rodentia; Retroviridae; Retina; MYO7A; Lentivirus; Genetic disease; Virus; Visual system; Eye disease; Vertebrata; Mammalia; Mouse; Usher syndrome; ENT disease; Gene therapy

One of the most disabling forms of retinal degeneration occurs in Usher syndrome, since it affects patients who already suffer from deafness. Mutations in the myosin VIIa gene (MYO7A) cause a major subtype of Usher syndrome, type 1B. Owing to the loss of function nature of Usher 1B and the relativel...