CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues.
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Salman A. et al, (2024), Int J Mol Sci, 25
Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?
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Bellingrath J-S. et al, (2023), Frontiers in Molecular Neuroscience, 16
A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery.
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Major L. et al, (2023), Int J Mol Sci, 24
Erratum: Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues.
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Chandler LC. et al, (2023), Mol Ther Methods Clin Dev, 28
Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases.
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Hansen S. et al, (2023), Cells, 12
Impaired glutamylation of RPGR ORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants
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Cehajic-Kapetanovic J. et al, (2022), Proceedings of the National Academy of Sciences, 119
Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.
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Staurenghi F. et al, (2022), Int J Mol Sci, 23
New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome.
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Major L. et al, (2022), Int J Mol Sci, 23
Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations.
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Salman A. et al, (2022), Pharmaceutics, 14
CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin
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Kaukonen M. et al, (2022), Genes, 13, 1327 - 1327
AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models.
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Wang J-H. et al, (2022), Sci Rep, 12
Envisioning the development of a CRISPR-Cas mediated base editing strategy for a patient with a novel pathogenic CRB1 single nucleotide variant.
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Bellingrath J-S. et al, (2022), Ophthalmic Genet, 1 - 10
Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids.
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McClements ME. et al, (2022), Transl Vis Sci Technol, 11
The Scope of Pathogenic ABCA4 Mutations Targetable by CRISPR DNA Base Editing Systems—A Systematic Review
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Piotter E. et al, (2022), Frontiers in Genetics, 12
Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
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Orlans HO. et al, (2021), Nature Communications, 12
In Silico Analysis of Pathogenic CRB1 Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention.
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Bellingrath J-S. et al, (2021), Genes (Basel), 12
CRISPR Systems Suitable for Single AAV Vector Delivery.
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Stevanovic M. et al, (2021), Curr Gene Ther
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
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Chandler LC. et al, (2021), Molecular Therapy - Methods & Clinical Development, 22, 52 - 65
Therapy Approaches for Stargardt Disease
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Piotter E. et al, (2021), Biomolecules, 11, 1179 - 1179
Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina
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Salman A. et al, (2021), Cells, 10, 1957 - 1957