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Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

CRISPR-CasRx effectively reduces ALS- and FTD-causing C9orf72 sense and antisense repeat derived RNAs and proteins in cell lines, patient iPSC-neurons and two independent mouse models of C9orf72 repeat expansion.

Liam Kempthorne
Deniz Vaizoglu
Adrian M. Isaacs
ResearchOpen Access08 Jan 2025
Nature Communications

Volume: 16, P: 1-15
Astrocyte-targeted gene delivery of interleukin 2 specifically increases brain-resident regulatory T cell numbers and protects against pathological neuroinflammation

Liston and colleagues design a gene-delivery system to specifically target astrocytes in the central nervous system to express IL-2 and thereby expand/maintain T_reg cells to suppress neuroinflammation.

Lidia Yshii
Emanuela Pasciuto
Adrian Liston
ResearchOpen Access26 May 2022
Nature Immunology

Volume: 23, P: 878-891

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Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

Astrocyte-targeted gene delivery of interleukin 2 specifically increases brain-resident regulatory T cell numbers and protects against pathological neuroinflammation

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