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

scientific article published on April 2016

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 is …
instance of (P31):
scholarly articleQ13442814

External links are
P356DOI10.1089/HUM.2015.142
P932PMC publication ID4840830
P698PubMed publication ID27036983

P50authorCamasamudram VijayasarathyQ130317925
Ronald A. BushQ130317927
Paul SievingQ62091603
P2093author name stringYong Zeng
Jinbo Li
Peter Colosi
Zhijian Wu
Suja Hiriyanna
Maria Santos
Sten Kjellstrom
P2860cites workRetinoschisin, the X-linked retinoschisis protein, is a secreted photoreceptor protein, and is expressed and released by Weri-Rb1 cellsQ22254690
Positional cloning of the gene associated with X-linked juvenile retinoschisisQ24317256
Sensing extracellular matrix: an update on discoidin domain receptor functionQ24318809
Inactivation of the murine X-linked juvenile retinoschisis gene, Rs1h, suggests a role of retinoschisin in retinal cell layer organization and synaptic structureQ24534221
Vision 1 year after gene therapy for Leber's congenital amaurosisQ24607981
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 yearQ24634801
Case 15-2009: a man with coma after cardiac arrestQ95802924
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkeyQ27329160
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialQ28262431
Effect of gene therapy on visual function in Leber's congenital amaurosisQ28277981
RS-1 Gene Delivery to an Adult Rs1h Knockout Mouse Model Restores ERG b-Wave with Reversal of the Electronegative Waveform of X-Linked RetinoschisisQ28506399
The mouse X-linked juvenile retinoschisis cDNA: expression in photoreceptorsQ30634873
Detecting outliers when fitting data with nonlinear regression - a new method based on robust nonlinear regression and the false discovery rateQ31033935
Expression of X-linked retinoschisis protein RS1 in photoreceptor and bipolar cellsQ32061912
A retinal neuronal developmental wave of retinoschisin expression begins in ganglion cells during layer formationQ33206204
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferQ33315634
Molecular Mechanisms Leading to Null-Protein Product from Retinoschisin (RS1) Signal-Sequence Mutants in X-Linked Retinoschisis (XLRS) DiseaseQ34349489
Correlation between SD-OCT, immunocytochemistry and functional findings in an animal model of retinal degeneration.Q34755346
Preclinical safety evaluation of a recombinant AAV8 vector for X-linked retinoschisis after intravitreal administration in rabbitsQ34768369
OUTER RETINAL TUBULATION IN ADVANCED AGE-RELATED MACULAR DEGENERATION: Optical Coherence Tomographic Findings Correspond to HistologyQ35772837
X-linked retinoschisis: RS1 mutation severity and age affect the ERG phenotype in a cohort of 68 affected male subjectsQ35826648
X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms.Q35907087
Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transferQ36040298
Congenital X-linked retinoschisis classification systemQ36583433
Synaptic pathology in retinoschisis knockout (Rs1-/y) mouse retina and modification by rAAV-Rs1 gene deliveryQ36915061
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kineticsQ36937189
Retinal morphological changes of patients with X-linked retinoschisis evaluated by Fourier-domain optical coherence tomographyQ37034082
Loss of retinoschisin (RS1) cell surface protein in maturing mouse rod photoreceptors elevates the luminance threshold for light-driven translocation of transducin but not arrestinQ37117364
Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouseQ37414094
Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approachQ37432588
The Status of RPE65 Gene Therapy Trials: Safety and EfficacyQ38337392
Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h-/- mouse.Q40362704
The major cell populations of the mouse retina.Q40838448
Coexpression and interaction of wild-type and missense RS1 mutants associated with X-linked retinoschisis: its relevance to gene therapyQ43523885
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisisQ43534316
Effect of late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouseQ44690695
Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: long-term rescue from retinal degenerationQ45869697
ISCEV Standard for full-field clinical electroretinography (2008 update).Q47217837
Retinal photoreceptor count, retinal surface area, and optic disc size in normal human eyesQ47378682
Photoreceptor and bipolar cell contributions to the cat electroretinogram: a kinetic model for the early part of the flash response.Q51577340
A schematic eye for the mouse, and comparisons with the ratQ70080416
The influence of MgCl2 and APB on the light-induced potassium changes and the ERG b-wave of the isolated superfused rat retinaQ71650249
Permeability and diffusion in vitreous humor: implications for drug deliveryQ74216293
P433issue5
P921main subjectgene therapyQ213901
X-linked retinoschisisQ3458131
P304page(s)376-389
P577publication date2016-04-01
P1433published inHuman Gene TherapyQ15757580
P1476titlePreclinical 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
P478volume27

Reverse relations

cites work (P2860)
Q97646319"Para-retinal" Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression
Q91903980AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration
Q55082904Analysis of Anatomic and Functional Measures in X-Linked Retinoschisis.
Q37676230Correction of Monogenic and Common Retinal Disorders with Gene Therapy
Q37715387Rearing Light Intensity Affects Inner Retinal Pathology in a Mouse Model of X-Linked Retinoschisis but Does Not Alter Gene Therapy Outcome
Q59353018Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery
Q89704563Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates
Q98726932Trans-Ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Transduction in Large Animal Eye After Intravitreal Vector Administration
Q58743510Translational Retinal Research and Therapies

Search more.