Work

Cas9 editing of ATXN1 in a spinocerebellar ataxia type 1 mice and human iPSC-derived neurons

Year: 2024

Type: article

Abstract: improved behavior deficits without increases in inflammatory markers. Importantly, the editing approach was confirmed in induced pluripotent stem cell (iPSC) neurons derived from patients with SCA1, promoting the translatability of the approach to patients.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2024.102317

Authors: Kelly J. Fagan, Guillem Chillon, Ellie M. Carrell, Elisa A. Waxman, Beverly L. Davidson

Institutions: Children's Hospital of Philadelphia, University of Pennsylvania

Language: English

Topic: Genetic Neurodegenerative Diseases

Subfield: Cellular and Molecular Neuroscience

Field: Neuroscience

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900