Work

Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells

Year: 2025

Type: article

Abstract: was achieved in patient-derived neuronal stem cells, showing the efficacy of this system in a disease-relevant cell type. This approach represents a novel therapeutic pathway for the treatment of HD.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2025.102561

Authors: Jennifer J. Waldo, Julian A.N.M. Halmai, Ankita Singh, Casiana E. Gonzalez, Yi-An Chen, Shaylyn A. Carthen, Jan A. Nolta, Kyle D. Fink

Institutions: University of California Davis Medical Center, UC Davis Health, University of California, Davis

Language: English

Topic: CRISPR and Genetic Engineering

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900

Funders: California Institute for Regenerative Medicine, California Institute of Regenerative Medicine, National Institute of Neurological Disorders and Stroke

Awards: G8511808479, MSCS ENGINEERED TO PRODUCE BDNF AND GENE EDITING CARGO FOR THE TREATMENT OF HUNTINGTON'S DISEASE, G919294750

OpenAlex ID: W4410352108

FWCI: 0.252

Cites: 71

Cited by: 1

Related to: 10