Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

@article{Maguire2009AgedependentEO,
  title={Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial},
  author={A. Maguire and K. High and A. Auricchio and J. F. Wright and E. Pierce and F. Testa and F. Mingozzi and J. Bennicelli and G. Ying and S. Rossi and A. Fulton and Kathleen A. Marshall and S. Banfi and D. Chung and Jessica I. W. Morgan and B. Hauck and O. Zelenaia and Xiaosong Zhu and L. Raffini and F. Coppieters and E. Baere and K. Shindler and N. Volpe and E. Surace and Carmela Acerra and A. Lyubarsky and T. Redmond and E. Stone and Junwei Sun and J. McDonnell and B. Leroy and F. Simonelli and J. Bennett},
  journal={The Lancet},
  year={2009},
  volume={374},
  pages={1597-1605}
}
BACKGROUND Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. We therefore did a phase 1 trial to assess the effect of gene therapy on retinal and visual function in children and adults with Leber's congenital amaurosis. METHODS We assessed the retinal and visual function in 12 patients (aged 8-44 years) with RPE65-associated Leber's congenital amaurosis given one subretinal injection of adeno… Expand
Long-term effect of gene therapy on Leber's congenital amaurosis.
Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy
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