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Publications

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 JS. et al, (2021), Genes, 12

CRISPR Systems Suitable for Single AAV Vector Delivery.

Stevanovic M. et al, (2021), Curr Gene Ther

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

Accurate Quantification of AAV Vector Genomes by Quantitative PCR.

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.

Dreismann AK. et al, (2021), Gene Ther

Genome-Editing Strategies for Treating Human Retinal Degenerations.

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.

Fry LE. et al, (2021), JAMA Ophthalmol

AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration

McClements ME. et al, (2021), BioMed Research International, 2021

An analysis of the Kozak consensus in retinal genes and its relevance to gene therapy.

McClements ME. et al, (2021), Mol Vis, 27, 233 - 242

CRISPR genome engineering for retinal diseases.

Kantor A. et al, (2021), 182, 29 - 79

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?

Han RC. et al, (2021), Expert Opin Orphan Drugs, 9, 13 - 24

The Scope of Pathogenic ABCA4 Mutations Targetable by CRISPR DNA Base Editing Systems-A Systematic Review.

Piotter E. et al, (2021), Front Genet, 12

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