Related references
Note: Only part of the references are listed.Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years
Samuel G. Jacobson et al.
ARCHIVES OF OPHTHALMOLOGY (2012)
Novel adeno-associated viral vectors for retinal gene therapy
L. H. Vandenberghe et al.
GENE THERAPY (2012)
The Human Rhodopsin Kinase Promoter in an AAV5 Vector Confers Rod- and Cone-Specific Expression in the Primate Retina
Shannon E. Boye et al.
HUMAN GENE THERAPY (2012)
Retinal Guanylyl Cyclase Isozyme 1 Is the Preferential In Vivo Target for Constitutively Active GCAP1 Mutants Causing Congenital Degeneration of Photoreceptors
Elena V. Olshevskaya et al.
JOURNAL OF NEUROSCIENCE (2012)
Restoration of Vision in the pde6β-deficient Dog, a Large Animal Model of Rod-cone Dystrophy
Lolita Petit et al.
MOLECULAR THERAPY (2012)
NMNAT1 mutations cause Leber congenital amaurosis
Marni J. Falk et al.
NATURE GENETICS (2012)
Mutations in NMNAT1 cause Leber congenital amaurosis and identify a new disease pathway for retinal degeneration
Robert K. Koenekoop et al.
NATURE GENETICS (2012)
AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia
Ji-Jing Pang et al.
PLOS ONE (2012)
Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa
William A. Beltran et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)
RPGR-Associated Retinal Degeneration in Human X-Linked RP and a Murine Model
Wei Chieh Huang et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2012)
Enzymatic Properties and Regulation of the Native Isozymes of Retinal Membrane Guanylyl Cyclase (RetGC) from Mouse Photoreceptors
Igor V. Peshenko et al.
BIOCHEMISTRY (2011)
Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency
Marija Mihelec et al.
HUMAN GENE THERAPY (2011)
AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa
Haoyu Mao et al.
HUMAN GENE THERAPY (2011)
Fighting Blindness with Adeno-Associated Virus Serotype 8
Alberto Auricchio
HUMAN GENE THERAPY (2011)
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy
Livia S. Carvalho et al.
HUMAN MOLECULAR GENETICS (2011)
The human visual cortex responds to gene therapy-mediated recovery of retinal function
Manzar Ashtari et al.
JOURNAL OF CLINICAL INVESTIGATION (2011)
XIAP Therapy Increases Survival of Transplanted Rod Precursors in a Degenerating Host Retina
Jingyu Yao et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)
IQCB1 Mutations in Patients with Leber Congenital Amaurosis
Alejandro Estrada-Cuzcano et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)
Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis
Xia Li et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)
Long-term Preservation of Cone Photoreceptors and Restoration of Cone Function by Gene Therapy in the Guanylate Cyclase-1 Knockout (GC1KO) Mouse
Sanford L. Boye et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)
Human CRB1-Associated Retinal Degeneration: Comparison with the rd8 Crb1-Mutant Mouse Model
Tomas S. Aleman et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)
A novel recessive GUCY2D mutation causing cone-rod dystrophy and not Leber's congenital amaurosis
Sibel A. Ugur Iseri et al.
EUROPEAN JOURNAL OF HUMAN GENETICS (2010)
Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
X. Sun et al.
GENE THERAPY (2010)
Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency
J. Pang et al.
GENE THERAPY (2010)
Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis
Basil S. Pawlyk et al.
HUMAN GENE THERAPY (2010)
Gene therapy rescues cone function in congenital achromatopsia
Andras M. Komaromy et al.
HUMAN MOLECULAR GENETICS (2010)
Novel functions of photoreceptor guanylate cyclases revealed by targeted deletion
Sukanya Karan et al.
MOLECULAR AND CELLULAR BIOCHEMISTRY (2010)
Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait
Alan F. Wright et al.
NATURE REVIEWS GENETICS (2010)
Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse
Shannon E. Boye et al.
PLOS ONE (2010)
Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78
Marina. S. Gorbatyuk et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)
Differential Macular Morphology in Patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-Related Leber Congenital Amaurosis
Sirichai Pasadhika et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2010)
Mutations in SPATA7 Cause Leber Congenital Amaurosis and Juvenile Retinitis Pigmentosa
Hui Wang et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2009)
Gene therapy for red-green colour blindness in adult primates
Katherine Mancuso et al.
NATURE (2009)
Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
William W. Hauswirth et al.
HUMAN GENE THERAPY (2008)
A Role for GCAP2 in Regulating the Photoresponse GUANYLYL CYCLASE ACTIVATION AND ROD ELECTROPHYSIOLOGY IN GUCA1B KNOCK-OUT MICE
Clint L. Makino et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2008)
Speed, spatial, and temporal tuning of rod and cone vision in mouse
Yumiko Umino et al.
JOURNAL OF NEUROSCIENCE (2008)
Safety and efficacy of gene transfer for Leber's congenital amaurosis
Albert M. Maguire et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W. B. Bainbridge et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
Artur V. Cideciyan et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Leber congenital amaurosis: Genes, proteins and disease mechanisms
Anneke I. den Hollander et al.
PROGRESS IN RETINAL AND EYE RESEARCH (2008)
AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEβ mutation
Ji-jing Pang et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2008)
Retinoschisin (RS1), the protein encoded by the X-linked retinoschisis gene, is anchored to the surface of retinal photoreceptor and bipolar cells through its interactions with a Na/K ATPase-SARM1 complex
Laurie L. Molday et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2007)
AAV-Mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter
Shahrokh C. Khani et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2007)
Clinical and molecular genetics of Leber's congenital amaurosis: A multicenter study of Italian patients
Francesca Simonelli et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2007)
Restoration of cone vision in a mouse model of achromatopsia
John J. Alexander et al.
NATURE MEDICINE (2007)
In vivo three-dimensional high-resolution imaging of rodent retina with spectral-domain optical coherence tomography
Marco Ruggeri et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2007)
The function of guanylate cyclase 1 and guanylate cyclase 2 in rod and cone photoreceptors
Wolfgang Baehr et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2007)
Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1
Shannon E. Haire et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2006)
Ca2+ and Mg2+ binding properties of GCAP-1 -: Evidence that Mg2+-bound form is the physiological activator of photoreceptor guanylyl cyclase
Igor V. Peshenko et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2006)
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
Samuel G. Jacobson et al.
MOLECULAR THERAPY (2006)
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis
JJ Pang et al.
MOLECULAR THERAPY (2006)
Beyond counting photons: Trials and trends in vertebrate visual transduction
ME Burns et al.
NEURON (2005)
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of X-linked juvenile retinoschisis
SH Min et al.
MOLECULAR THERAPY (2005)
Independent visual threshold measurements in the two eyes of freely moving rats and mice using a virtual-reality optokinetic system
RM Douglas et al.
VISUAL NEUROSCIENCE (2005)
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success
SG Jacobson et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
The Y99C mutation in guanylyl cyclase-activating protein 1 increases intracellular Ca2+ and causes photoreceptor degeneration in transgenic mice
EV Olshevskaya et al.
JOURNAL OF NEUROSCIENCE (2004)
Leber congenital amaurosis: Comprehensive survey of the genetic heterogeneity, refinement of the clinical definition, and genotype-phenotype correlations as a strategy for molecular diagnosis
S Hanein et al.
HUMAN MUTATION (2004)
Clinicopathologic effects of mutant GUCY2D in Leber congenital amaurosis
AH Milam et al.
OPHTHALMOLOGY (2003)
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
S Zolotukhin et al.
METHODS (2002)
Applications of the Morris water maze in the study of learning and memory
R D'Hooge et al.
BRAIN RESEARCH REVIEWS (2001)
Share Paper
