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CRISPR Interference–Potential Application in Retinal Disease

Journal article

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

RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences

Journal article

Fry LE. et al, (2020), International Journal of Molecular Sciences, 21, 777 - 777

Molecular Strategies for RPGR Gene Therapy.

Journal article

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

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

Journal article

Chandler LC. et al, (2019), Molecular Therapy - Methods & Clinical Development, 14, 77 - 89

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

An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4−/− Mice

Journal article

McClements ME. et al, (2019), Human Gene Therapy, 30, 590 - 600

A Quantitative Chloride Channel Conductance Assay for Efficacy Testing of AAV.BEST1.

Journal article

Wood SR. et al, (2019), Hum Gene Ther Methods, 30, 44 - 52

A Novel Achromatopsia Mouse Model Resulting From a Naturally Occurring Missense Change in Cngb3.

Journal article

Hassall MM. et al, (2018), Invest Ophthalmol Vis Sci, 59, 6102 - 6110

A Novel Achromatopsia Mouse Model Resulting From a Naturally Occurring Missense Change in Cngb3

Journal article

(2018), Investigative Ophthalmology and Visual Science

Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes

Journal article

McClements ME. and MacLaren RE., (2017), Yale Journal of Biology and Medicine

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina

Journal article

Hickey DG. et al, (2017), Gene Therapy, 24, 787 - 800

Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy

Journal article

De Silva SR. et al, (2017), Proceedings of the National Academy of Sciences, 114, 11211 - 11216

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina

Journal article

Patrício MI. et al, (2017), Molecular Therapy - Nucleic Acids, 6, 198 - 208

INVESTIGATION OF THE RELATIONSHIP BETWEEN LEVELS OF CHM GENE EXPRESSION AND RATE OF DISEASE PROGRESSION IN CHOROIDEREMIA

Conference paper

Patricio MI. et al, (2017), EUROPEAN JOURNAL OF OPHTHALMOLOGY, 27, E86 - E86

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