Tissue-Specific Effects of the DNA Helicase FANCJ/BRIP1/BACH1 on Repeat Expansion in a Mouse Model of the Fragile X-Related Disorders DOI Open Access
Diego Antonio Jimenez,

Alexandra K. Walker,

Karen Usdin

и другие.

International Journal of Molecular Sciences, Год журнала: 2025, Номер 26(6), С. 2655 - 2655

Опубликована: Март 15, 2025

Fragile X-related disorders (FXDs) are caused by the expansion of a CGG repeat tract in 5’-UTR FMR1 gene. The mechanism is likely shared with 45+ other human diseases resulting from expansion, process that has been shown to require key mismatch repair (MMR) factors. FANCJ, DNA helicase involved unwinding unusual secondary structures, implicated number processes including MMR. To test role FANCJ we crossed FancJ-null mice an FXD mouse model. We found loss resulted trend towards more extensive was significant for small intestine and male germline. This finding interesting implications raises possibility helicases may be important modifiers risk certain cell types.

Язык: Английский

Advances in Huntington’s Disease Biomarkers: A 10-Year Bibliometric Analysis and a Comprehensive Review DOI Creative Commons
Sarah Aqel, Jamil Ahmad,

Iman Saleh

и другие.

Biology, Год журнала: 2025, Номер 14(2), С. 129 - 129

Опубликована: Янв. 26, 2025

Neurodegenerative disorders (NDs) cause progressive neuronal loss and are a significant public health concern, with NDs projected to become the second leading global of death within two decades. Huntington’s disease (HD) is rare, ND caused by an autosomal-dominant mutation in huntingtin (HTT) gene, severe brain resulting debilitating motor, cognitive, psychiatric symptoms. Given complex pathology HD, biomarkers essential for performing early diagnosis, monitoring progression, evaluating treatment efficacy. However, identification consistent HD challenging due prolonged premanifest stage, HD’s heterogeneous presentation, its multiple underlying biological pathways. This study involves 10-year bibliometric analysis biomarker research, revealing key research trends gaps. The also features comprehensive literature review emerging biomarkers, concluding need better stratification patients well-designed longitudinal studies validate biomarkers. Promising candidate wet biomarkers— including neurofilament light chain protein (NfL), microRNAs, mutant HTT protein, specific metabolic inflammatory markers— discussed, emphasis on their potential utility stage. Additionally, reflecting structural deficits motor or behavioral impairments, such as neurophysiological (e.g., tapping, speech, EEG, event-related potentials) imaging MRI, PET, diffusion tensor imaging) evaluated. findings underscore that discovery validation reliable urgently require improved patient studies. Reliable particularly crucial optimizing clinical management strategies, enabling personalized approaches, advancing trials HD-modifying therapies.

Язык: Английский

Процитировано

0

Tissue-Specific Effects of the DNA Helicase FANCJ/BRIP1/BACH1 on Repeat Expansion in a Mouse Model of the Fragile X-Related Disorders DOI Open Access
Diego Antonio Jimenez,

Alexandra K. Walker,

Karen Usdin

и другие.

International Journal of Molecular Sciences, Год журнала: 2025, Номер 26(6), С. 2655 - 2655

Опубликована: Март 15, 2025

Fragile X-related disorders (FXDs) are caused by the expansion of a CGG repeat tract in 5’-UTR FMR1 gene. The mechanism is likely shared with 45+ other human diseases resulting from expansion, process that has been shown to require key mismatch repair (MMR) factors. FANCJ, DNA helicase involved unwinding unusual secondary structures, implicated number processes including MMR. To test role FANCJ we crossed FancJ-null mice an FXD mouse model. We found loss resulted trend towards more extensive was significant for small intestine and male germline. This finding interesting implications raises possibility helicases may be important modifiers risk certain cell types.

Язык: Английский

Процитировано

0