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Anson Cheng

As seen in: Nature, Stem Cell Reports, Initium Media
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Articles by Anson Cheng

AAV8- PDE6B with 3’UTR provides rescue of photoreceptor structure and function in the rd10 mouse model of retinitis pigmentosa

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By Anson Cheng, Leszek Lisowski, John Grigg, Ian Alexander, Robyn Jamieson, Steven S. Eamegdool, Amin Sabri, Fidelle Chahine Karam
| Nature Verified
Abstract Retinitis pigmentosa (RP), a leading cause of inherited blindness, results in a subset of cases from phosphodiesterase 6 (PDE6) deficiency. We evaluated a novel gene therapy construct, AAV8-RK-PDE6B-3′UTR, in the rd10 mouse model which has a homozygous missense variant that disrupts the function of the β-subunit of PDE6.
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Connecting cilium, stress response, and proteostasis abnormalities inform variant and therapy assessment in RPGRIP1 retinal organoids

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By Anson Cheng, Hani Kim, Milan Fernando, Benjamin Nash, Nader Aryamanesh, John Grigg, Pengyi Yang, Anai Gonzalez-Cordero, Robyn Jamieson
| Stem Cell Reports Verified
Results We applied TruSight One Clinical Exome sequencing to examine a bioinformatic gene panel of 65 IRD genes, which identified RPGRIP1 variants in 2 patients, LCA-1 and LCA-2, with poor vision and nystagmus manifested in the first 3 months of life. Electroretinograms showed reduced responses, and poor visual acuity was consistent at last review at ages 29 and 27 years for LCA-1 and LCA-2, respectively, with ophthalmic changes characteristic of LCA (Figures S1A and S1B).
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By Anson Cheng
| Initium Media Verified
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