Safety and efficacy of gene transfer for Leber's congenital amaurosis.
By A. Maguire, F. Simonelli, Eric A. Pierce, E. Pugh, F. Mingozzi, J. Bennicelli, S. Banfi, Kathleen A. Marshall, F. Testa, E. Surace and 22 more. New England Journal of Medicine, 2008
2,123 citations recorded by the source corpus.
Subfields: Molecular Biology, Ophthalmology, Radiology, Nuclear Medicine and Imaging
View Paper: https://www.semanticscholar.org/paper/5b8920390a573eea6569f840579b0c66409eb418
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In Molecular Biology
- N. Bertrand
- Xiaoyang Xu
- Joshua T. Mendell
- O. Farokhzad
- M. Kay
- P. Vivekanandan
- Eric M. Pridgen
- Hun-Way Hwang
In Ophthalmology
In Radiology, Nuclear Medicine and Imaging
Citations in the corpus
- Retinitis Pigmentosa: Genes and Disease Mechanisms (2011, background): “Results in pre-clinical studies have recently led to three encouraging gene therapy clinical trials in which patients affected by LCA were sub-retinal injected with recombinant adeno-associated viral vectors (rAAV) containing the human RPE65 cDNA [24-26].”
- 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].”
- Therapeutic delivery of miR-26a inhibits cancer cell proliferation and induces tumor-specific apoptosis (2009, positive): “Finally, the general safety of AAV has been well documented, with clinical trials using this platform already under way (Carter, 2005; Maguire et al., 2008; Park et al., 2008).”
- Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics (2008, background): “S3), contrary to an observation made in another trial (16).”
- 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”