Publications by authors named "Elizabeth M Simpson"

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Establishing Preclinical Quantitative Parameters for Future Assessment of Corneal and Retinal Therapeutics for Aniridia.
Sif G Kaad, Andrea J Korecki, Elizabeth M Simpson

Invest Ophthalmol Vis Sci· July 2025

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Escape from X-chromosome inactivation at KDM5C is driven by promoter-proximal DNA elements and enhanced by domain context.
Samantha Peeters, Sarah Baldry, Andrea J Korecki, Aditi Srinivasan, Wyeth W Wasserman, Elizabeth M Simpson

Hum Mol Genet· May 2025

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Minimally Humanized Exon-18 Mouse Cell Lines Validate Preclinical CRISPR/Cas9 Approach to Treat Weaver Syndrome.
William T Gibson, Tess C Lengyell, Andrea J Korecki, Sanne M Janssen, Bethany A Adair, Elizabeth M Simpson

Hum Gene Ther· March 2025

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New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates.
Alissandra de Moura Gomes, Terri L Petkau, Andrea J Korecki, Oriol Fornes, Adriana Galvan, Elizabeth M Simpson

Sci Rep· November 2024

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Taf1 knockout is lethal in embryonic male mice and heterozygous females show weight and movement disorders.
Elisa M Crombie, Andrea J Korecki, Karen Cleverley, Bethany A Adair, Thomas J Cunningham, Elizabeth M Simpson

Dis Model Mech· July 2024

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An oocyte-specific Cas9-expressing mouse for germline CRISPR/Cas9-mediated genome editing.
Denise G Lanza, Jianqiang Mao, Isabel Lorenzo, Lan Liao, John R Seavitt, Elizabeth M Simpson

Genesis· April 2024

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Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression.
Rikke Hahn Kofoed, Elizabeth M Simpson, Kullervo Hynynen, Isabelle Aubert

Gene Ther· December 2023

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rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality.
Seyedeh Zeinab Mirjalili Mohanna, Andrea J Korecki, Elizabeth M Simpson

Gene Ther· September 2023

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The GLP-1 agonist, exendin-4, stimulates LH secretion in female sheep.
Elizabeth M Simpson, Iain J Clarke, Christopher J Scott, Cyril P Stephen, Alexandra Rao

J Endocrinol· September 2023

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ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons.
Bethany A Adair, Andrea J Korecki, Diana Djaksigulova, Pamela K Wagner, Nina Y Chiu, Elizabeth M Simpson

Ophthalmol Ther· August 2023

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OnTarget: in silico design of MiniPromoters for targeted delivery of expression.
Oriol Fornes, Tamar V Av-Shalom, Andrea J Korecki, Rachelle A Farkas, David J Arenillas, Elizabeth M Simpson

Nucleic Acids Res· July 2023

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A novel association between platelet filamin A and soluble N-ethylmaleimide sensitive factor attachment proteins regulates granule secretion.
Kalyan Golla, Manoj Paul, Tess C Lengyell, Elizabeth M Simpson, Hervé Falet

Res Pract Thromb Haemost· May 2023

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LNP-mediated delivery of CRISPR RNP for wide-spread in vivo genome editing in mouse cornea.
Seyedeh Zeinab Mirjalili Mohanna, Diana Djaksigulova, Austin M Hill, Pamela K Wagner, Elizabeth M Simpson

J Control Release· October 2022

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Targeted Expression of Retinoschisin by Retinal Bipolar Cells in XLRS Promotes Resolution of Retinoschisis Cysts Sans RS1 From Photoreceptors.
Camasamudram Vijayasarathy, Yong Zeng, Dario Marangoni, Lijin Dong, Zhuo-Hua Pan, Elizabeth M Simpson

Invest Ophthalmol Vis Sci· October 2022

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Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis.
Rikke Hahn Kofoed, Stefan Heinen, Joseph Silburt, Sonam Dubey, Chinaza Lilian Dibia, Elizabeth M Simpson

Mol Ther Methods Clin Dev· December 2021

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Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells.
Andrea J Korecki, Jorge L Cueva-Vargas, Oriol Fornes, Jessica Agostinone, Rachelle A Farkas, Elizabeth M Simpson

Gene Ther· June 2021

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Human progranulin-expressing mice as a novel tool for the development of progranulin-modulating therapeutics.
Terri L Petkau, Benjamin Life, Ge Lu, Jasmine Yang, Oriol Fornes, Elizabeth M Simpson

Neurobiol Dis· June 2021

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Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System.
Adriana Galvan, Terri L Petkau, Austin M Hill, Andrea J Korecki, Ge Lu, Elizabeth M Simpson

Hum Gene Ther· June 2021

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A novel mouse model for pyridoxine-dependent epilepsy due to antiquitin deficiency.
Hilal H Al-Shekaili, Terri L Petkau, Izabella Pena, Tess C Lengyell, Nanda M Verhoeven-Duif, Elizabeth M Simpson

Hum Mol Genet· November 2020

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Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia.
Seyedeh Zeinab Mirjalili Mohanna, Jack W Hickmott, Siu Ling Lam, Nina Y Chiu, Tess C Lengyell, Elizabeth M Simpson

Mol Ther Methods Clin Dev· June 2020

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Ferroportin-mediated iron export from vascular endothelial cells in retina and brain.
Bailey H Baumann, Wanting Shu, Ying Song, Elizabeth M Simpson, Samira Lakhal-Littleton

Exp Eye Res· October 2019

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Correction: Epistasis between Pax6 and genetic background reinforces the value of defined hybrid mouse models for therapeutic trials.
Jack W Hickmott, Uvini Gunawardane, Kimberly Jensen, Andrea J Korecki, Elizabeth M Simpson

Gene Ther· April 2019

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Twenty-Seven Tamoxifen-Inducible iCre-Driver Mouse Strains for Eye and Brain, Including Seventeen Carrying a New Inducible-First Constitutive-Ready Allele.
Andrea J Korecki, Jack W Hickmott, Siu Ling Lam, Lisa Dreolini, Anthony Mathelier, Elizabeth M Simpson

Genetics· April 2019

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How do genes that escape from X-chromosome inactivation contribute to Turner syndrome?
Samantha B Peeters, Andrea J Korecki, Sarah E L Baldry, Christine Yang, Kira Tosefsky, Elizabeth M Simpson

Am J Med Genet C Semin Med Genet· March 2019

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Epistasis between Pax6 and genetic background reinforces the value of defined hybrid mouse models for therapeutic trials.
Jack W Hickmott, Uvini Gunawardane, Kimberly Jensen, Andrea J Korecki, Elizabeth M Simpson

Gene Ther· December 2018

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