Spotlight on childhood blindness
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Reference24 articles.
1. Leber Congenital Amaurosis–A Model for Efficient Genetic Testing of Heterogeneous Disorders: LXIV Edward Jackson Memorial Lecture
2. Leber congenital amaurosis: Genes, proteins and disease mechanisms
3. Mutations in the CEP290 (NPHP6) Gene Are a Frequent Cause of Leber Congenital Amaurosis
4. A Novel Locus for Leber Congenital Amaurosis Maps to Chromosome 6q
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1. Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing;Molecular Therapy - Nucleic Acids;2023-03
2. Prime Editing for Human Gene Therapy: Where Are We Now?;Cells;2023-02-07
3. Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases;Nature Biomedical Engineering;2021-08-26
4. Therapeutic adenine base editing corrects nonsense mutation and improves visual function in a mouse model of Leber congenital amaurosis;2021-01-08
5. Knocking out lca5 in zebrafish causes cone-rod dystrophy due to impaired outer segment protein trafficking;Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease;2019-10
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