Pages that link to "Q30537764"
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The following pages link to Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years (Q30537764):
Displaying 50 items.
- NMNAT1 mutations cause Leber congenital amaurosis (Q24863444) (← links)
- Clinical development of gene therapy: results and lessons from recent successes (Q26743393) (← links)
- Retinal dystrophies, genomic applications in diagnosis and prospects for therapy (Q26770321) (← links)
- Gene therapy: progress and predictions (Q26772109) (← links)
- Recent advances and future prospects in choroideremia (Q26773223) (← links)
- Concise Review: Patient-Specific Stem Cells to Interrogate Inherited Eye Disease (Q26774786) (← links)
- Adeno-associated Virus as a Mammalian DNA Vector (Q26782605) (← links)
- Regenerative medicine for the special senses: restoring the inputs (Q27000739) (← links)
- Promising and delivering gene therapies for vision loss (Q27007079) (← links)
- Gene therapy of inherited retinal degenerations: prospects and challenges (Q27009261) (← links)
- Immunology of AAV-Mediated Gene Transfer in the Eye (Q27023474) (← links)
- A comprehensive review of retinal gene therapy (Q27023914) (← links)
- Adeno-associated virus-mediated cancer gene therapy: current status (Q27026177) (← links)
- Müller glia activation in response to inherited retinal degeneration is highly varied and disease-specific (Q27311456) (← links)
- Canine CNGA3 Gene Mutations Provide Novel Insights into Human Achromatopsia-Associated Channelopathies and Treatment (Q27316578) (← links)
- CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome) (Q27333793) (← links)
- Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium (Q27694625) (← links)
- Development of gene and stem cell therapy for ocular neurodegeneration (Q28081549) (← links)
- Review: the history and role of naturally occurring mouse models with Pde6b mutations (Q28304672) (← links)
- Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. (Q28306059) (← links)
- Targeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating Peptide (Q28394174) (← links)
- Exciting directions in glaucoma (Q28542397) (← links)
- Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012 (Q28708928) (← links)
- Caspase inhibition with XIAP as an adjunct to AAV vector gene-replacement therapy: improving efficacy and prolonging the treatment window (Q28729935) (← links)
- AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia (Q28730645) (← links)
- Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial (Q30277652) (← links)
- The effect of injection speed and serial injection on propidium iodide entry into cultured HeLa and primary neonatal fibroblast cells using lance array nanoinjection. (Q30378182) (← links)
- Macular function in macular degenerations: repeatability of microperimetry as a potential outcome measure for ABCA4-associated retinopathy trials (Q30512453) (← links)
- The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. (Q30525669) (← links)
- Assessment of Rod, Cone, and Intrinsically Photosensitive Retinal Ganglion Cell Contributions to the Canine Chromatic Pupillary Response (Q30834824) (← links)
- rAAV-mediated subcellular targeting of optogenetic tools in retinal ganglion cells in vivo (Q31120899) (← links)
- Pupillary Light Reflexes in Severe Photoreceptor Blindness Isolate the Melanopic Component of Intrinsically Photosensitive Retinal Ganglion Cells. (Q32186770) (← links)
- Progress of stem/progenitor cell-based therapy for retinal degeneration (Q33655064) (← links)
- Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration (Q33729610) (← links)
- An unparalleled engine for discovery and clinical introduction: the clinical and translational science awards and gene therapy (Q33751443) (← links)
- Towards a quantitative OCT image analysis (Q33754203) (← links)
- Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies (Q33832987) (← links)
- Rescue of enzymatic function for disease-associated RPE65 proteins containing various missense mutations in non-active sites (Q33846472) (← links)
- Long-Term Safety of Transplanting Human Bone Marrow Stromal Cells into the Extravascular Spaces of the Choroid of Rabbits (Q33862445) (← links)
- Fiber-modified adenovirus for central nervous system Parkinson's disease gene therapy (Q34102305) (← links)
- A high content screening approach to identify molecules neuroprotective for photoreceptor cells (Q34139442) (← links)
- Poly(β-amino ester)-nanoparticle mediated transfection of retinal pigment epithelial cells in vitro and in vivo (Q34281984) (← links)
- Human cone visual pigment deletions spare sufficient photoreceptors to warrant gene therapy (Q34373332) (← links)
- Advantage of Whole Exome Sequencing over Allele-Specific and Targeted Segment Sequencing in Detection of Novel TULP1 Mutation in Leber Congenital Amaurosis (Q34404974) (← links)
- Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosis (Q34406815) (← links)
- The impact of cHS4 insulators on DNA transposon vector mobilization and silencing in retinal pigment epithelium cells (Q34461198) (← links)
- Blue cone monochromacy: visual function and efficacy outcome measures for clinical trials (Q34473512) (← links)
- Improvement and decline in vision with gene therapy in childhood blindness. (Q34474831) (← links)
- Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease (Q34497784) (← links)
- The golden era of ocular disease gene discovery: race to the finish (Q34556960) (← links)