Elizabeth M. Simpson
Vancouver, BC V6H0B3, Canada
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 45 publications
Humanized mice carrying a pathogenic GRN deletion as a pre-clinical platform for targeted gene therapies in frontotemporal dementia.
Journal: Neurobiology of disease
Published: March 06, 2026
First pilot study of intrastromal rAAV-PAX6 gene therapy suggests improved corneal thickness and transcription correction in aniridic mouse.
Journal: Molecular therapy. Advances
Published: November 13, 2025
First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia.
Journal: Gene therapy
Published: August 13, 2025
Establishing Preclinical Quantitative Parameters for Future Assessment of Corneal and Retinal Therapeutics for Aniridia.
Journal: Investigative ophthalmology & visual science
Published: July 23, 2025
Escape from X-chromosome inactivation at KDM5C is driven by promoter-proximal DNA elements and enhanced by domain context.
Journal: Human molecular genetics
Published: December 09, 2024
Taf1 knockout is lethal in embryonic male mice and heterozygous females show weight and movement disorders.
Journal: Disease models & mechanisms
Published: February 01, 2024
An oocyte-specific Cas9-expressing mouse for germline CRISPR/Cas9-mediated genome editing.
Journal: Genesis (New York, N.Y. : 2000)
Published: December 22, 2023
OnTarget: in silico design of MiniPromoters for targeted delivery of expression.
Journal: Nucleic acids research
Published: March 28, 2023
ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons.
Journal: Ophthalmology and therapy
Published: March 12, 2023
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality.
Journal: Gene therapy
Published: January 06, 2023
Last Updated: 09/26/2026