McCallister, Tristan X.
Lim, Colin K. W.
Singh, Mayuri
Zhang, Sijia
Ahsan, Najah S.
Terpstra, William M.
Xiong, Alisha Y. https://orcid.org/0009-0000-3269-3081
Zeballos C, M. Alejandra https://orcid.org/0000-0002-5194-2909
Powell, Jackson E.
Drnevich, Jenny https://orcid.org/0000-0002-1401-8311
Kang, Yifei
Gaj, Thomas https://orcid.org/0000-0001-6004-9664
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (1U01NS122102-01A1, 1R01NS123556-01A1)
U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (5R01GM141296)
Muscular Dystrophy Association (MDA602798)
Amyotrophic Lateral Sclerosis Association (20-IIP-516)
Article History
Received: 5 December 2023
Accepted: 11 December 2024
First Online: 8 January 2025
Competing interests
: T.G. and J.P. are inventors on a pending patent application entitled, “Therapeutic gene silencing with CRISPR-Cas13” (18/576,407) filed by the University of Illinois Urbana-Champaign, which involves the AAV-mediated delivery of RfxCas13d to the brain and spinal cord for gene silencing-based applications. T.G., T.X.M., and C.L. are inventors on a pending provisional patent application entitled, “Targeting the G<sub>4</sub>C<sub>2</sub> repeat-containing with a high-fidelity CRISPR-Cas13 system improves abnormalities associated with ALS/FTD” (63/605,767) filed by the University of Illinois Urbana-Champaign, which involves methods for implementing RfxCas13d for suppressing the repeat RNAs for C9ORF72-linked ALS/FTD. The remaining authors declare no competing interests.