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

By A. Maguire, K. High, A. Auricchio, J. F. Wright, Eric A. Pierce, F. Testa, F. Mingozzi, J. Bennicelli, Gui-shuang Ying, S. Rossi and 24 more. The Lancet, 2009

816 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology, Ophthalmology

View Paper: https://www.semanticscholar.org/paper/ae099dcf7c470b629a578077bf16e152afa2ea96

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Genetics

1. M. Kay

In Molecular Biology

1. N. Bertrand
2. Xiaoyang Xu
3. O. Farokhzad
4. M. Kay
5. Eric M. Pridgen
6. Xue-Qing Zhang
7. A. Swami
8. Robert Langer

Citations in the corpus

• Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years (2011, background): “In subgroup analyses, VA change in foveal-fixating study eyes or in extrafoveal-fixating study and control eyes did not achieve statistical significance (P= .07, .17, and .56, respectively); counterintuitively, foveal-fixating control eyes showed a significant (P=.02) improvement in VA.”
• Interactions of nanomaterials and biological systems: implications to personalized nanomedicine (2012, background): “In this perspective, scientists at the Children’s Hospital of Philadelphia have invested tremendous efforts in developing an adenovirus-based treatment for Leber Congenital Amaurosis (LCA), a very rare degenerative disease which irremediably leads to blindness [227-229].”
• Gene therapy clinical trials worldwide to 2012 – an update (2013, result): “Importantly, improvements in both objective and subjective measures of vision have been reported andmaintained for up to 2 years [36,37], supporting further clinical trials for this and other forms of LCA.”
• State-of-the-art gene-based therapies: the road ahead (2011, background): “In this early Phase I study, there was a marked improvement in measurable vision parameters as well as restoration of ambulatory vision in some patient”