CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues.
Journal article
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.
Journal article
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.
Journal article
Chandler LC. et al, (2023), Mol Ther Methods Clin Dev, 28
Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases.
Journal article
Hansen S. et al, (2023), Cells, 12
Programmable RNA editing with endogenous ADAR enzymes - a feasible option for the treatment of inherited retinal disease?
Journal article
Bellingrath J-S. et al, (2023), Front Mol Neurosci, 16
Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.
Journal article
Staurenghi F. et al, (2022), Int J Mol Sci, 23
New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome.
Journal article
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.
Journal article
Salman A. et al, (2022), Pharmaceutics, 14
AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models.
Journal article
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.
Journal article
Bellingrath J-S. et al, (2022), Ophthalmic Genet, 1 - 10
Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids.
Journal article
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.
Journal article
Bellingrath J-S. et al, (2021), Genes (Basel), 12
CRISPR Systems Suitable for Single AAV Vector Delivery.
Journal article
Stevanovic M. et al, (2021), Curr Gene Ther
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina.
Journal article
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.
Journal article
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
Conference paper
Yusuf IH. et al, (2021), MOLECULAR THERAPY, 29, 68 - 69
Accurate Quantification of AAV Vector Genomes by Quantitative PCR.
Journal article
Martinez-Fernandez de la Camara C. et al, (2021), Genes (Basel), 12
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells.
Journal article
Dreismann AK. et al, (2021), Gene Ther
Genome-Editing Strategies for Treating Human Retinal Degenerations.
Journal article
Quinn J. et al, (2021), Hum Gene Ther, 32, 247 - 259
Analysis of Pathogenic Variants Correctable With CRISPR Base Editing Among Patients With Recessive Inherited Retinal Degeneration.
Journal article
Fry LE. et al, (2021), JAMA Ophthalmol