Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy

COMMENTED

Retinal Diseases

Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease

JAMA Ophthalmol.

Johnson S, Buessing M, O'Connell T, Pitluck S, Ciulla TA

doi: 10.1001/

Retinal Diseases

Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level

Sci Rep.

Udry F, Decembrini S, Gamm DM, Déglon N, Kostic C, Arsenijevic Y

doi: 10.1038/s41598-020-65657-y.

Retinal Diseases

The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients

Ophthalmology.

Ashtari M, Nikonova ES, Marshall KA, Young GJ, Aravand P, Pan W, Ying GS, Willett AE, Mahmoudian M, Maguire AM, Bennett J

doi: 10.1016/j.ophtha.2017.01.029.

Retinal Diseases

Clinically Focused Molecular Investigation of 1000 Consecutive Families with Inherited Retinal Disease

Ophthalmology.

Stone EM, Andorf JL, Whitmore SS, DeLuca AP, Giacalone JC, Streb LM, Braun TA, Mullins RF, Scheetz TE, Sheffield VC, Tucker BA

doi: 10.1016/j.ophtha.

Retinal Diseases

Retinal vessel oximetry in children with inherited retinal diseases

Acta Ophthalmol.

Della Volpe Waizel M, Scholl HPN, Valmaggia C, Todorova MG

doi: 10.1111/aos.14466.

Gene therapy in optic nerve disease

Curr Opin Ophthalmol.

DeBusk A, Moster ML

doi: 10.1097/ICU.

Macular DiseasesRetinal Diseases

Technique of retinal gene therapy: delivery of viral vector into the subretinal space

Eye (Lond).

Xue K, Groppe M, Salvetti AP, MacLaren RE

doi: 10.1038/eye.2017.158.

Blindness and Vision Impairment

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

Ophthalmology.

Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, Wright JF, High KA, Bennett J

doi: 10.1016/j.ophtha.2019.06.017.

Retinal Diseases

Genetic Basis of Inherited Retinal Disease in a Molecularly Characterized Cohort of More Than 3000 Families from the United Kingdom

Ophthalmology.

Pontikos N, Arno G, Jurkute N, Schiff E, Ba-Abbad R, Malka S, Gimenez A, Georgiou M, Wright G, Armengol M, Knight H, Katz M, Moosajee M, Yu-Wai-Man P, Moore AT, Michaelides M, Webster AR, Mahroo OA

doi: 10.1016/j.ophtha.2020.04.008.

Related content

Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease

Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level

The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients

Clinically Focused Molecular Investigation of 1000 Consecutive Families with Inherited Retinal Disease

Retinal vessel oximetry in children with inherited retinal diseases

Gene therapy in optic nerve disease

Technique of retinal gene therapy: delivery of viral vector into the subretinal space

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

Genetic Basis of Inherited Retinal Disease in a Molecularly Characterized Cohort of More Than 3000 Families from the United Kingdom