Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

By T. Coelho, D. Adams, Ana Silva, P. Lozeron, P. Hawkins, T. Mant, J. Pérez, Joseph Chiesa, S. Warrington, E. Tranter and 21 more. New England Journal of Medicine, 2013

902 citations recorded by the source corpus.

Subfields: Cell Biology, Molecular Biology, Oncology

View Paper: https://www.semanticscholar.org/paper/2d700ec14c4baa30bafdb09896939ce3a9b74dac

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In Cell Biology

1. O. Farokhzad
2. R. Falk

In Molecular Biology

1. Vikram Agarwal
2. David P. Bartel
3. N. Bertrand
4. Xiaoyang Xu
5. M. Stoffel
6. O. Farokhzad
7. M. Hope
8. Rémy Denzler

In Oncology

1. O. Farokhzad
2. Michael S. Diamond

Citations in the corpus

• Modified mRNA vaccines protect against Zika virus infection (2017, positive): “We selected LNPs for delivery of the modified mRNA as they have been validated in clinical trials for siRNA delivery and are well tolerated compared to other non-viral delivery systems (Coelho et al., 2013).”
• Nonbiopsy Diagnosis of Cardiac Transthyretin Amyloidosis (2016, background): “Thus, there is a major unmet need to diagnose and characterize cardiac amyloidosis at the earliest opportunity, noting that cardiac ATTR amyloidosis in particular is probably greatly underdiagnosed and fast becoming a treatable cause of heart failure.(18,19) Radionuclide bone scintigraphy with techn”
• Wild-type transthyretin amyloidosis as a cause of heart failure with preserved ejection fraction. (2015, background): “Moreover, several new generation drugs have been proven effective to treat hereditary TTR amyloidosis in phase 2 and 3 studies.(10,11) Although data on cardiac involvement are lacking, some of these drugs have shown preliminary favourable results and might be effective also to treat HFpEF caused by ”
• Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis (2018, background): “IIIB: walking with two sticks or crutches 11 (14) 28 (19) 39 (17)”
• Lipid Nanoparticle Systems for Enabling Gene Therapies. (2017, background): “A major limitation of viral vectors is the induction of neutralizing antibodies that prevent repeat administrations, a problem that has not been observed to-date for LNP systems delivering siRNA.(27) As with the application of this platform in vaccines, we predict examples of its utility in the gene”
• Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines (2019, positive): “14 Our own vaccine trials with MC3-based LNPs for in fl uenza gave 100% seroconversion with a 100-m g dose of modi fi ed mRNA.”