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Baughn et al., 2023 - Google Patents

Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies

Baughn et al., 2023

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Document ID
8570406911377728081
Author
Baughn M
Melamed Z
López-Erauskin J
Beccari M
Ling K
Zuberi A
Presa M
Gonzalo-Gil E
Maimon R
Vazquez-Sanchez S
Chaturvedi S
Bravo-Hernández M
Taupin V
Moore S
Artates J
Acks E
Ndayambaje I
Agra de Almeida Quadros A
Jafar-Nejad P
Rigo F
Bennett C
Lutz C
Lagier-Tourenne C
Cleveland D
Publication year
Publication venue
Science

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Loss of nuclear TDP-43 is a hallmark of neurodegeneration in TDP-43 proteinopathies, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP-43 mislocalization results in cryptic splicing and polyadenylation of pre–messenger RNAs (pre …
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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