Gene augmentation therapy for a missense substitution in the cGMP-binding domain of ovine CNGA3 gene restores vision in day-blind sheep

Keyphrases

• Achromatopsia 30%
• Adeno-associated Viral Vector 10%
• Amino Acid Substitution 10%
• Awassi Sheep 10%
• Behavioral Assessment 10%
• Bioinformatics Analysis 10%
• Causative mutation 40%
• CNGA3 100%
• Complementary DNA (cDNA) 10%
• Concordance Test 10%
• Cone Photoreceptor 10%
• Cyclic GMP 100%
• Day Blindness 10%
• Deep Sequencing 10%
• Deleteriousness 10%
• Gene Augmentation Therapy 100%
• Genetic Complementation 10%
• Genomic Regions 10%
• Heterozygote 20%
• Histologic Examination 10%
• Histological Techniques 10%
• Human Genes 10%
• Immunohistochemical Examination 10%
• Impaired Vision 10%
• Lamb 20%
• Large Animal Model 10%
• Ligand-binding Domain 100%
• Missense 10%
• Missense Substitution 100%
• Newborn Lambs 10%
• Nonsynonymous 10%
• Novel Form 10%
• Novel mutation 10%
• Orthologs 20%
• Ovine 100%
• Photopic Response 10%
• Photopic Vision 10%
• Premature Termination 10%
• Sequence Analysis 10%
• Sequence Reads 10%
• Sheep 100%
• Subretinal Delivery 10%
• Visual Impairment 10%

Biochemistry, Genetics and Molecular Biology

• Achromatopsia 100%
• Adeno Associated Virus Vector 33%
• Amino Acid Substitution 33%
• Animal Model 33%
• Binding Domain 100%
• Bioinformatics 33%
• Cyclic Guanosine Monophosphate 100%
• Deep Sequencing 33%
• Genetic Complementation 33%
• Genetics 33%
• Genomics 33%
• Guanosine Monophosphate 33%
• Lamb 100%
• Missense 100%
• Photopic Vision 33%
• Photoreceptor Cell 33%