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Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction.
- Published in:
- Pharmaceutics, 2023, v. 15, n. 2, p. 322, doi. 10.3390/pharmaceutics15020322
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- Article
Effective delivery of large genes to the retina by dual AAV vectors.
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- EMBO Molecular Medicine, 2014, v. 6, n. 2, p. 194, doi. 10.1002/emmm.201302948
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- Article
RPE-Directed Gene Therapy Improves Mitochondrial Function in Murine Dry AMD Models.
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- International Journal of Molecular Sciences, 2023, v. 24, n. 4, p. 3847, doi. 10.3390/ijms24043847
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- Article
SARM1 Ablation Is Protective and Preserves Spatial Vision in an In Vivo Mouse Model of Retinal Ganglion Cell Degeneration.
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- International Journal of Molecular Sciences, 2022, v. 23, n. 3, p. 1606, doi. 10.3390/ijms23031606
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- Article
A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors.
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- Frontiers in Neuroscience, 2017, p. 1, doi. 10.3389/fnins.2017.00521
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- Article
Gene therapies for inherited retinal disorders.
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- Visual Neuroscience, 2014, v. 31, n. 4/5, p. 289, doi. 10.1017/S0952523814000133
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- Article
microRNA regulatory circuits in a mouse model of inherited retinal degeneration.
- Published in:
- Scientific Reports, 2016, p. 31431, doi. 10.1038/srep31431
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- Publication type:
- Article
Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy.
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- European Journal of Human Genetics, 2013, v. 21, n. 1, p. 62, doi. 10.1038/ejhg.2012.112
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- Article
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement.
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- European Journal of Human Genetics, 2011, v. 19, n. 10, p. 1074, doi. 10.1038/ejhg.2011.86
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- Article
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement.
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- 2011
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- Correction Notice
RNAi of COL1A1 in mesenchymal progenitor cells.
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- European Journal of Human Genetics, 2004, v. 12, n. 10, p. 864, doi. 10.1038/sj.ejhg.5201230
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- Article
Optimized OPA1 Isoforms 1 and 7 Provide Therapeutic Benefit in Models of Mitochondrial Dysfunction.
- Published in:
- Frontiers in Neuroscience, 2020, v. 14, p. N.PAG, doi. 10.3389/fnins.2020.571479
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- Publication type:
- Article
AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas.
- Published in:
- Frontiers in Neuroscience, 2020, v. 14, p. N.PAG, doi. 10.3389/fnins.2020.00891
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- Publication type:
- Article
Non-photoreceptor Expression of Tulp1 May Contribute to Extensive Retinal Degeneration in Tulp1-/- Mice.
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- Frontiers in Neuroscience, 2020, v. 14, p. 1, doi. 10.3389/fnins.2020.00656
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- Article
AAV‐mediated gene therapy improving mitochondrial function provides benefit in age‐related macular degeneration models.
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- Clinical & Translational Medicine, 2022, v. 12, n. 8, p. 1, doi. 10.1002/ctm2.952
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- Article
A Mutation-Independent Therapeutic Strategy for Dominant Dystrophic Epidermolysis Bullosa.
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- Journal of Investigative Dermatology, 2013, v. 133, n. 12, p. 2793, doi. 10.1038/jid.2013.241
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- Publication type:
- Article
AAV-mediated chronic over-expression of SNAP-25 in adult rat dorsal hippocampus impairs memory-associated synaptic plasticity.
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- Journal of Neurochemistry, 2010, v. 112, n. 4, p. 991, doi. 10.1111/j.1471-4159.2009.06516.x
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- Article
Novel 199 base pair NEFH promoter drives expression in retinal ganglion cells.
- Published in:
- Scientific Reports, 2020, v. 10, n. 1, p. N.PAG, doi. 10.1038/s41598-020-73257-z
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- Publication type:
- Article
Toward an elucidation of the molecular genetics of inherited retinal degenerations.
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- Human Molecular Genetics, 2017, v. 26, p. R2, doi. 10.1093/hmg/ddx185
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- Article
Development of strategies for conditional RNA interference.
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- Journal of Gene Medicine, 2007, v. 9, n. 4, p. 287, doi. 10.1002/jgm.1018
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- Article
Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa.
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- Molecular Therapy, 2011, v. 19, n. 4, p. 642, doi. 10.1038/mt.2010.293
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- Publication type:
- Article
Improved Retinal Function in a Mouse Model of Dominant Retinitis Pigmentosa Following AAV-delivered Gene Therapy.
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- Molecular Therapy, 2009, v. 17, n. 4, p. 593, doi. 10.1038/mt.2008.301
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- Publication type:
- Article
1019. Development of Tissue-Specific RNAi for Gene Therapy.
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- Molecular Therapy, 2006, v. 13, p. S392, doi. 10.1016/j.ymthe.2006.08.1114
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- Publication type:
- Article
1020. AAV Delivery of Suppression and Replacement Constructs for Rhodopsin-Linked Autosomal Dominant Retinitis Pigmentosa<sup>*</sup>.
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- Molecular Therapy, 2006, v. 13, p. S392, doi. 10.1016/j.ymthe.2006.08.1115
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- Publication type:
- Article
1021. Suppression and Replacement Strategies for Rhodopsin-Linked Autosomal Dominant Retinitis Pigmentosa<sup>*</sup>.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S393, doi. 10.1016/j.ymthe.2006.08.1116
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- Publication type:
- Article
Toward a Gene Therapy for Dominant Disease: Validation of an RNA Interference-Based Mutation-Independent Approach
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- Molecular Therapy, 2005, v. 12, n. 3, p. 555, doi. 10.1016/j.ymthe.2005.03.028
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- Article
RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture.
- Published in:
- Human Mutation, 2006, v. 27, n. 3, p. 260, doi. 10.1002/humu.20287
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- Article
Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10).
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- Human Molecular Genetics, 2008, v. 17, n. 14, p. 2084, doi. 10.1093/hmg/ddn107
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- Article
Murine model of autosomal dominant retinitis pigmentosa generated by targeted deletion at codon 307 of the rds–peripherin gene.
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- Human Molecular Genetics, 2002, v. 11, n. 9, p. 1005, doi. 10.1093/hmg/11.9.1005
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- Publication type:
- Article
Identification of an IMPDH1 mutationin autosomal dominant retinitis pigmentosa (RP10) revealed followingcomparative microarray analysis of transcripts derived from retinasof wild-type and Rho–/– mice.
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- Human Molecular Genetics, 2002, v. 11, n. 5, p. 547, doi. 10.1093/hmg/11.5.547
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- Article
AAV-NDI1 Therapy Provides Significant Benefit to Murine and Cellular Models of Glaucoma.
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- International Journal of Molecular Sciences, 2024, v. 25, n. 16, p. 8876, doi. 10.3390/ijms25168876
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- Article
Strategems in Vitro for Gene Therapies Directed to Dominant Mutations.
- Published in:
- Human Molecular Genetics, 1997, v. 6, n. 9, p. 1415, doi. 10.1093/hmg/6.9.1415
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- Article