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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

Filtration of Short-Wavelength Light Provides Therapeutic Benefit in Retinitis Pigmentosa Caused by a Common Rhodopsin Mutation.

Journal article

Orlans HO. et al, (2019), Invest Ophthalmol Vis Sci, 60, 2733 - 2742

Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia.

Journal article

Xue K. et al, (2018), Nat Med, 24, 1507 - 1512

Differential roles for cryptochromes in the mammalian retinal clock.

Journal article

Wong JCY. et al, (2018), FASEB J, 32, 4302 - 4314

Choroideremia: molecular mechanisms and development of AAV gene therapy.

Journal article

Patrício MI. et al, (2018), Expert Opin Biol Ther, 18, 807 - 820

Vector shedding and immunology results from a gene therapy clinical trial for choroideremia

Conference paper


The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured by In Vitro Prenylation of RAB6A.

Journal article

Patrício MI. et al, (2018), Mol Ther Methods Clin Dev, 9, 288 - 295

A missense mutation in Katnal1 underlies behavioural, neurological and ciliary anomalies.

Journal article

Banks G. et al, (2018), Mol Psychiatry, 23, 713 - 722

Vector Shedding and Immunogenicity Sampling for Retinal Gene Therapy.

Journal article

Barnard AR. et al, (2018), Methods Mol Biol, 1715, 359 - 371

Gene Therapy for Color Blindness.

Journal article

Hassall MM. et al, (2017), Yale J Biol Med, 90, 543 - 551

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), Proc Natl Acad Sci U S A, 114, 11211 - 11216

Prenylation of Rab6a as a Potency Assay for Choroideremia Gene Therapy

Journal article

Patricio MI. et al, (2017), MOLECULAR THERAPY, 25, 142 - 143

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