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Accurate Quantification of AAV Vector Genomes by Quantitative PCR.

Journal article

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

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

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

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

Journal article

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

Genome editing strategies for treating human retinal degenerations.

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Quinn J. et al, (2020), Hum Gene Ther

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 Ophthalmol

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

Journal article

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

CRISPR Interference-Potential Application in Retinal Disease.

Journal article

Peddle CF. et al, (2020), Int J Mol Sci, 21

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues.

Journal article

Chandler LC. et al, (2019), Mol Ther Methods Clin Dev, 14, 77 - 89

Molecular Strategies for RPGR Gene Therapy.

Journal article

Cehajic Kapetanovic J. et al, (2019), Genes (Basel), 10

AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization.

Journal article

Hughes CP. et al, (2019), Mol Ther Methods Clin Dev, 13, 86 - 98

Clinical and Molecular Characterization of PROM1-Related Retinal Degeneration.

Journal article

Cehajic-Kapetanovic J. et al, (2019), JAMA Netw Open, 2

The Location of Exon 4 Mutations in RP1 Raises Challenges for Genetic Counseling and Gene Therapy.

Journal article

Nanda A. et al, (2019), Am J Ophthalmol, 202, 23 - 29

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