E
Elena Miranda
Researcher at Sapienza University of Rome
Publications - 110
Citations - 4887
Elena Miranda is an academic researcher from Sapienza University of Rome. The author has contributed to research in topics: Neuroserpin & Familial encephalopathy with neuroserpin inclusion bodies. The author has an hindex of 32, co-authored 98 publications receiving 4453 citations. Previous affiliations of Elena Miranda include United States Geological Survey & California State University.
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Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells
Kosuke Yusa,S. Tamir Rashid,Helene Strick-Marchand,Helene Strick-Marchand,Ignacio Varela,Pei-Qi Liu,David Paschon,Elena Miranda,Elena Miranda,Adriana Ordóñez,Nicholas R.F. Hannan,Foad J. Rouhani,Foad J. Rouhani,Sylvie Darche,Sylvie Darche,Graeme J.M. Alexander,Stefan J. Marciniak,Noemi Fusaki,Mamoru Hasegawa,Michael C. Holmes,James P. Di Santo,James P. Di Santo,David A. Lomas,Allan Bradley,Ludovic Vallier +24 more
TL;DR: This work shows that a combination of zinc finger nucleases (ZFNs) and piggyBac technology in human iPSCs can achieve biallelic correction of a point mutation in the α1-antitrypsin (A1AT, also known as SERPINA1) gene that is responsible for α1
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Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells
S. Tamir Rashid,Sébastien Corbineau,Nicholas R.F. Hannan,Stefan J. Marciniak,Elena Miranda,Graeme J.M. Alexander,Isabel Huang-Doran,Julian L. Griffin,Lars Ährlund-Richter,Jeremy N. Skepper,Robert K. Semple,Anne Weber,David A. Lomas,Ludovic Vallier +13 more
TL;DR: These patient-derived hepatocytes demonstrate that it is possible to model diseases whose phenotypes are caused by pathological dysregulation of key processes within adult cells, and a simple and effective platform for hepatocyte generation from patient-specific human iPS cells.
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Endoplasmic reticulum dysfunction in neurological disease
Benoit D. Roussel,Benoit D. Roussel,Antonina J. Kruppa,Elena Miranda,Damian C. Crowther,David A. Lomas,Stefan J. Marciniak +6 more
TL;DR: By targeting components of these signalling responses, amelioration of their toxic effects and so the treatment of a range of neurodegenerative disorders might become possible.
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Intraneuronal Aβ, non-amyloid aggregates and neurodegeneration in a Drosophila model of Alzheimer’s disease
Damian C. Crowther,Kerri J. Kinghorn,Elena Miranda,Richard M. Page,J.A. Curry,F.A.I. Duthie,David Gubb,David A. Lomas +7 more
TL;DR: This model demonstrates that intracellular accumulation and non-amyloid aggregates of Aβ are sufficient to cause the neurodegeneration of Alzheimer’s disease and provides a platform to dissect the pathways of neurodegenersation in Alzheimer's disease and to develop novel therapeutic interventions.
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A cyclic peptide inhibitor of HIF-1 heterodimerization that inhibits hypoxia signaling in cancer cells
Elena Miranda,Ida Karin Nordgren,Abigail L. Male,Charlotte E. Lawrence,Franciane Hoakwie,F. Cuda,William Court,Keith R. Fox,Paul A. Townsend,Graham Packham,Suzanne A. Eccles,Ali Tavassoli +11 more
TL;DR: Cyclo-CLLFVY, identified from a library of 3.2 million cyclic hexapeptides using a genetically encoded high-throughput screening platform, is reported as an inhibitor of the HIF-1α/Hif-1β protein–protein interaction in vitro and in cells.