Sophie R. Thomson

Edinburgh, SCT EH89XD, United Kingdom

Last Updated: 09/26/2026

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Found 8 publications

AAV9-Mediated PTEN Gene Therapy Rescues Hepatic Pathology and Neuroanatomical Abnormalities in a Murine Model of PTEN Hamartoma Tumour Syndrome.

AAV9-Mediated PTEN Gene Therapy Rescues Hepatic Pathology and Neuroanatomical Abnormalities in a Murine Model of PTEN Hamartoma Tumour Syndrome.

Journal: Pharmaceutics

Published: June 03, 2026

Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome.

Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome.

Journal: Science translational medicine

Published: April 02, 2025

Editorial: Recent advances in mechanisms and therapeutics for Fragile X Syndrome and autism.

Editorial: Recent advances in mechanisms and therapeutics for Fragile X Syndrome and autism.

Journal: Frontiers in neuroscience

Published: March 16, 2023

Excess ribosomal protein production unbalances translation in a model of Fragile X Syndrome.

Excess ribosomal protein production unbalances translation in a model of Fragile X Syndrome.

Journal: Nature communications

Published: September 27, 2021

Cell-Type-Specific Translation Profiling Reveals a Novel Strategy for Treating Fragile X Syndrome.

Cell-Type-Specific Translation Profiling Reveals a Novel Strategy for Treating Fragile X Syndrome.

Journal: Neuron

Published: December 06, 2016

Bioenergetic status modulates motor neuron vulnerability and pathogenesis in a zebrafish model of spinal muscular atrophy.

Bioenergetic status modulates motor neuron vulnerability and pathogenesis in a zebrafish model of spinal muscular atrophy.

Journal: PLoS genetics

Published: November 08, 2016

Morphological characteristics of motor neurons do not determine their relative susceptibility to degeneration in a mouse model of severe spinal muscular atrophy.

Morphological characteristics of motor neurons do not determine their relative susceptibility to degeneration in a mouse model of severe spinal muscular atrophy.

Journal: PloS one

Published: October 05, 2012

Using induced pluripotent stem cells (iPSC) to model human neuromuscular connectivity: promise or reality?

Using induced pluripotent stem cells (iPSC) to model human neuromuscular connectivity: promise or reality?

Journal: Journal of anatomy

Published: December 03, 2011

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Last Updated: 09/26/2026