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Therapy Approaches for Stargardt Disease

Journal article

Piotter E. et al, (2021), Biomolecules, 11, 1179 - 1179

Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina

Journal article

Salman A. et al, (2021), Cells, 10, 1957 - 1957

Accurate Quantification of AAV Vector Genomes by Quantitative PCR

Journal article

Martinez-Fernandez de la Camara C. et al, (2021), Genes, 12, 601 - 601

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

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McClements ME. et al, (2021), BioMed Research International, 2021, 1 - 8

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

Journal article

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

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

Journal article

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

CRISPR genome engineering for retinal diseases.


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

Genome-Editing Strategies for Treating Human Retinal Degenerations

Journal article

Quinn J. et al, (2020), Human Gene Therapy

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances

Journal article

McClements ME. et al, (2020), Frontiers in Neuroscience, 14

Association of a Novel Intronic Variant in RPGR With Hypomorphic Phenotype of X-Linked Retinitis Pigmentosa

Journal article

Cehajic-Kapetanovic J. et al, (2020), JAMA Ophthalmology

CRISPR-Cas9 DNA Base-Editing and Prime-Editing.

Journal article

Kantor A. et al, (2020), Int J Mol Sci, 21

Analysis of Early Cone Dysfunction in an In Vivo Model of Rod-Cone Dystrophy

Journal article

Hassall MM. et al, (2020), International Journal of Molecular Sciences, 21, 6055 - 6055

Promoter Orientation within an AAV-CRISPR Vector Affects Cas9 Expression and Gene Editing Efficiency

Journal article

Fry LE. et al, (2020), The CRISPR Journal, 3, 276 - 283

Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy

Journal article

Chandler L. et al, (2020), International Journal of Molecular Sciences, 21, 4972 - 4972

Assessment of AAV Dual Vector Safety in the Abca4−/− Mouse Model of Stargardt Disease

Journal article

McClements ME. et al, (2020), Translational Vision Science & Technology, 9, 20 - 20

CRISPR Interference–Potential Application in Retinal Disease

Journal article

Peddle CF. et al, (2020), International Journal of Molecular Sciences, 21, 2329 - 2329

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