Genetic therapies and potential therapeutic applications of CRISPR activators in the eye.
Ng BW. et al, (2024), Prog Retin Eye Res
Gene Therapies in Clinical Development to Treat Retinal Disorders.
McClements ME. et al, (2024), Mol Diagn Ther
Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration.
Yusuf IH. et al, (2024), Mol Ther
CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues.
Salman A. et al, (2024), Int J Mol Sci, 25
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
Programmable RNA editing with endogenous ADAR enzymes - a feasible option for the treatment of inherited retinal disease?
Bellingrath J-S. et al, (2023), Front Mol Neurosci, 16
Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.
Staurenghi F. et al, (2022), Int J Mol Sci, 23
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
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
In Silico Analysis of Pathogenic CRB1 Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention.
Bellingrath J-S. et al, (2021), Genes (Basel), 12
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina.
Chandler LC. et al, (2021), Mol Ther Methods Clin Dev, 22, 52 - 65
Deep phenotyping of the Cdhr1-/- mouse validates its use in pre-clinical studies for human CDHR1-associated retinal degeneration.
Yusuf IH. et al, (2021), Exp Eye Res, 208
Gene Therapy Rescues Cone and Rod Function in a Pre-Clinical Model of CDHR1-Associated Retinal Degeneration through Restoration of Photoreceptor Outer Segments
Yusuf IH. et al, (2021), MOLECULAR THERAPY, 29, 68 - 69