Engineering Duplex RNAs for Challenging Targets: Recognition of GGGGCC/CCCCGG Repeats at the ALS/FTD C9orf72 Locus-Reference-Cited by-同舟云学术

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Engineering Duplex RNAs for Challenging Targets: Recognition of GGGGCC/CCCCGG Repeats at the ALS/FTD C9orf72 Locus

Published:2015-11 Issue:11 Volume:22 Page:1505-1511
ISSN:1074-5521
Container-title:Chemistry & Biology
language:en
Short-container-title:Chemistry & Biology

Author:

Hu Jiaxin,Liu Jing,Li Liande,Gagnon Keith T.,Corey David R.

Funder

NIH

ALS Association

Robert A. Welch Foundation

Publisher

Elsevier BV

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology,Molecular Biology,Molecular Medicine,General Medicine,Biochemistry

Reference20 articles.

1. Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS;DeJesus-Hernandez;Neuron,2012

2. RNA toxicity from ALS/FTD C9ORF72 expansion is mitigated by antisense intervention;Donnelly;Neuron,2013

3. Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats;Fiszer;Nucleic Acids Res.,2011

4. Antisense transcripts of the expanded C9ORF72 hexanucleotide repeat form nuclear RNA foci and undergo repeat associated non-ATG translation in c9FTD/ALS;Gendron;Acta Neuropathol.,2013

5. C9orf72 nucleotide repeat structures initiate molecular cascades of disease;Haeusler;Nature,2014

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1. Therapeutic reduction of GGGGCC repeat RNA levels by hnRNPA3 suppresses neurodegeneration in Drosophila models of C9orf72-linked ALS/FTD;Human Molecular Genetics;2023-01-15

2. Efficient Cloning and Sequence Validation of Repetitive and High GC-Content Short Hairpin RNAs;Human Gene Therapy;2022-08-01

3. Gene Therapy in Amyotrophic Lateral Sclerosis;Cells;2022-06-29

4. Gene therapy for ALS: A review;Molecular Therapy;2021-12

5. Insights into disease mechanisms and potential therapeutics for C9orf72-related amyotrophic lateral sclerosis/frontotemporal dementia;Ageing Research Reviews;2020-12

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