RNAi-Based Treatment for Neovascular Age-Related Macular Degeneration by Sirna-027

Kaiser, Peter K.; Symons, R.C. Andrew; Shah, Syed Mahmood; Quinlan, Edward; Tabandeh, Homayoun; Diana, V.; Reisen, Gail; Lockridge, Jennifer A.; Short, Brian; Guerciolini, Roberto; Nguyen, Quan Dong

doi:10.1016/j.ajo.2010.02.006

Cited by 157 publications

(95 citation statements)

References 14 publications

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“…Unlike other siRNAs that have been evaluated for the treatment of neovascular AMD, 14 PF-04523655 is a 19-nucleotide, methylated double-stranded siRNA specifically targeting the RTP801 gene. In addition to its inhibition of the RTP801 gene, the methylation, double stranding and short length of PF-04523655 may facilitate cellular entry and prevent its degradation in vivo.…”

Section: Introductionmentioning

confidence: 99%

Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients

Nguyen

Schachar

Nduaka

et al. 2012

Eye

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Section: Introductionmentioning

confidence: 99%

Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients

Nguyen

Schachar

Nduaka

et al. 2012

Eye

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“…It shows promise in treating previously untreatable or difficult to treat diseases and has the potential to be used prophylactically and therapeutically. Clinical trials are ongoing for conditions including cancer, neovascular age-related macular degeneration (AMD) [5] and respiratory viral infections. Concerns over off-target effects, immunogenicity and toxicity remain due to the adverse effects witnessed during previous gene therapy trials [6].…”

Section: Rna Interference In Respiratory Diseasementioning

confidence: 99%

‘Smart‘ non-viral Delivery Systems for Targeted Delivery of RNAi to the Lungs

Ramsey

Hibbitts

Barlow

et al. 2012

Therapeutic Delivery

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SummaryThe emergence of RNA interference (RNAi) offers a potentially exciting new therapeutic paradigm for respiratory diseases. However effective delivery remains a key requirement for their translation into the clinic and has been a major factor in the limited clinical success seen to-date. Inhalation offers tissue-specific targeting of the RNAi to treat respiratory diseases and a diminished risk of off-target effects. In order to deliver RNAi directly to the respiratory tract via inhalation "smart" non-viral carriers are required to protect the RNAi during delivery/aerosolisation and enhance cell-specific uptake to target cells. Herein, we review the state of the art in therapeutic aerosol bioengineering (TAB) and specifically non-viral siRNA delivery platforms for delivery via inhalation. This includes developments in inhaler device engineering and particle engineering including manufacturing methods and excipients used in TAB that underpin the development of "smart" cell-type specific delivery systems to target siRNA to respiratory epithelial cells and/or alveolar macrophages.

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“…The targeted genes for CNV treatment are mostly VEGF and VEGF receptors (90)(91)(92). The silencing of hypoxia inducible factor-1alpha (HIF-1alpha), that regulates the VEGF expression in hypoxic conditions of ocular angiogenesis is also under investigation to treat CNV (93).…”

Section: Small Interference Rna (Sirna)mentioning

confidence: 99%