scholarly journals Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function

2016 ◽  
Vol 27 (5) ◽  
pp. 376-389 ◽  
Author(s):  
Ronald A. Bush ◽  
Yong Zeng ◽  
Peter Colosi ◽  
Sten Kjellstrom ◽  
Suja Hiriyanna ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Dose Escalation ◽  
Structure And Function ◽  
Dose Escalation Study ◽  
Retinal Structure ◽  
And Function ◽  
Dose Dependent

scholarly journals Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration

2020 ◽  
Vol 18 ◽  
pp. 582-594
Author(s):  
Scott H. Greenwald ◽  
Emily E. Brown ◽  
Michael J. Scandura ◽  
Erin Hennessey ◽  
Raymond Farmer ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Retinal Degeneration ◽  
Structure And Function ◽  
Retinal Structure ◽  
And Function

XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma

Gene Therapy ◽  
2021 ◽  
Author(s):  
Shagana Visuvanathan ◽  
Adam N. Baker ◽  
Pamela S. Lagali ◽  
Stuart G. Coupland ◽  
Garfield Miller ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Ganglion Cell ◽  
Retinal Ganglion Cell ◽  
Cell Structure ◽  
Structure And Function ◽  
Retinal Ganglion ◽  
And Function

scholarly journals Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models

2015 ◽  
Vol 24 (11) ◽  
pp. 3104-3118 ◽  
Author(s):  
Lucie P. Pellissier ◽  
Peter M. Quinn ◽  
C. Henrique Alves ◽  
Rogier M. Vos ◽  
Jan Klooster ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
Mouse Models ◽  
Glial Cells ◽  
Structure And Function ◽  
Müller Glial Cells ◽  
Retinal Structure ◽  
And Function

scholarly journals Gene therapy via canalostomy approach preserves auditory and vestibular functions in a mouse model of Jervell and Lange-Nielsen syndrome type 2

2021 ◽  
Vol 12 (1) ◽  
Author(s):  
Xuewen Wu ◽  
Li Zhang ◽  
Yihui Li ◽  
Wenjuan Zhang ◽  
Jianjun Wang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Inner Ear ◽  
Viral Vectors ◽  
Survival Rates ◽  
Semicircular Canals ◽  
Dependent Manner ◽  
Syndrome Type ◽  
Dose Dependent

AbstractMutations in voltage-gated potassium channel KCNE1 cause Jervell and Lange-Nielsen syndrome type 2 (JLNS2), resulting in congenital deafness and vestibular dysfunction. We conducted gene therapy by injecting viral vectors using the canalostomy approach in Kcne1−/− mice to treat both the hearing and vestibular symptoms. Results showed early treatment prevented collapse of the Reissner’s membrane and vestibular wall, retained the normal size of the semicircular canals, and prevented the degeneration of inner ear cells. In a dose-dependent manner, the treatment preserved auditory (16 out of 20 mice) and vestibular (20/20) functions in mice treated with the high-dosage for at least five months. In the low-dosage group, a subgroup of mice (13/20) showed improvements only in the vestibular functions. Results supported that highly efficient transduction is one of the key factors for achieving the efficacy and maintaining the long-term therapeutic effect. Secondary outcomes of treatment included improved birth and litter survival rates. Our results demonstrated that gene therapy via the canalostomy approach, which has been considered to be one of the more feasible delivery methods for human inner ear gene therapy, preserved auditory and vestibular functions in a dose-dependent manner in a mouse model of JLNS2.

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