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Your search keyword '"Boye, Shannon E."' showing total 7 results
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7 results on '"Boye, Shannon E."'

5. Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study

Author

Yang, Paul, Pardon, Laura P, Ho, Allen C, Lauer, Andreas K, Yoon, Dan, Boye, Shannon E, Boye, Sanford L, Roman, Alejandro J, Wu, Vivian, Garafalo, Alexandra V, Sumaroka, Alexander, Swider, Malgorzata, Viarbitskaya, Iryna, Aleman, Tomas S, Pennesi, Mark E, Kay, Christine N, Fujita, Kenji P, and Cideciyan, Artur V

Abstract

Leber congenital amaurosis 1 (LCA1), caused by mutations in GUCY2D, is a rare inherited retinal disease that typically causes blindness in early childhood. The aim of this study was to evaluate the safety and preliminary efficacy of ascending doses of ATSN-101, a subretinal AAV5 gene therapy for LCA1.

Published
2024
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6. Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.

Author

Duncan JL, Bowman A, Laster A, Gelfman C, Birch DG, Boye SE, Daiger SP, Del Priore L, Zack DJ, and Handa JT

Subjects
Humans, Genetic Therapy methods, Geographic Atrophy genetics, Geographic Atrophy therapy, cis-trans-Isomerases genetics, Macular Degeneration genetics, Macular Degeneration therapy, Macular Degeneration pathology, Retinal Degeneration genetics, Retinal Degeneration therapy
Abstract

Inherited retinal degeneration (IRD) disease and age-related macular degeneration (AMD) are leading causes of irreversible vision loss and blindness. Although significant progress has advanced the field in the past 5 years, significant challenges remain. The current article reviews the accomplishments and research advances that have fueled the development of treatments for patients with IRD and AMD, including the first approved gene-augmentation treatment for RPE65-related retinal degeneration and complement inhibition therapies to slow progression of geographic atrophy (GA) in AMD. The article outlines opportunities to address gaps and unmet needs that should lead to additional progress toward the development of treatments for patients with IRDs and non-neovascular AMD in the future.

Published
2024
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