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[This corrects the article DOI: 10.3389/fvets.2025.1550617.].
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http://dx.doi.org/10.3389/fvets.2025.1668896 | DOI Listing |
Front Vet Sci
August 2025
[This corrects the article DOI: 10.3389/fvets.2025.
View Article and Find Full Text PDFMol Ther Nucleic Acids
September 2025
Department of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia.
Loss-of-function mutations in the gene cause β-catenin deficiency, resulting in CTNNB1 syndrome, a rare neurodevelopmental disorder characterized by motor and cognitive impairments. Given the wide variety of mutations across and its dosage sensitivity, a mutation-independent therapeutic approach that preserves endogenous gene regulation is critically needed. This study introduces spliceosome-mediated RNA -splicing as a novel approach to restore β-catenin production.
View Article and Find Full Text PDFFamilial dysautonomia (FD) is a rare autosomal recessive neurodegenerative disorder caused by a splicing mutation in the gene. It predominantly affects the sensory and autonomic nervous systems, with progressive vision loss due to optic neuropathy being a universal and debilitating symptom. Retinal pathology in FD involves progressive thinning of the retinal nerve fiber layer (RNFL), resulting from the degeneration of retinal ganglion cells (RGCs).
View Article and Find Full Text PDFCell Death Differ
September 2025
Department of Urology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Nat Commun
August 2025
Laboratory of Translational Genomics, DCEG, National Cancer Institute, Rockville, MD, USA.