A comparison study of pathological features and drug efficacy between Drosophila models of C9orf72 ALS/FTD.

ABSTRACT: Amyotrophic lateral sclerosis is a devastating neurodegenerative disease with a complex genetic basis, presenting both in familial and sporadic forms. The hexanucleotide (G₄C₂) repeat expansion in the C9orf72 gene, which triggers distinct pathogenic mechanisms, has been identified as a major contributor to familial and sporadic Amyotrophic lateral sclerosis cases. Animal models have proven pivotal in understanding these mechanisms; however, discrepancies between models due to variable transgene sequence, expression levels, and toxicity profiles complicate the translation of findings. Herein, we provide a systematic comparison of 7 publicly available Drosophila transgenes modeling the G₄C₂ expansion under uniform conditions, evaluating variations

SUBMITTER: Lee D 

PROVIDER: S-EPMC10880080 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature