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Publications

Genetic therapies and potential therapeutic applications of CRISPR activators in the eye.

Ng BW. et al, (2024), Prog Retin Eye Res, 102

Single-cell transcriptomic analysis of retinal immune regulation and blood-retinal barrier function during experimental autoimmune uveitis

Quinn J. et al, (2024), Scientific Reports, 14

CRISPR targeting of SNPs associated with Age-related Macular Degeneration in ARPE-19 cells: a potential model for manipulating the complement system

Salman A. et al, (2024)

Gene Therapies in Clinical Development to Treat Retinal Disorders

McClements ME. et al, (2024), Molecular Diagnosis & Therapy

Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration

Yusuf IH. et al, (2024), Molecular Therapy, 32, 1445 - 1460

CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues

Salman A. et al, (2024), International Journal of Molecular Sciences, 25, 1697 - 1697

Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?

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.

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.

Chandler LC. et al, (2023), Mol Ther Methods Clin Dev, 28

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases.

Hansen S. et al, (2023), Cells, 12

Impaired glutamylation of RPGR ORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants

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

Staurenghi F. et al, (2022), International Journal of Molecular Sciences, 23, 11673 - 11673

New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome.

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

Salman A. et al, (2022), Pharmaceutics, 14, 1842 - 1842

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin

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.

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.

Bellingrath J-S. et al, (2022), Ophthalmic Genet, 1 - 10

Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids.

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

Piotter E. et al, (2022), Frontiers in Genetics, 12

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

Orlans HO. et al, (2021), Nature Communications, 12

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