BR112022020412A2 - Sequências antissenso para tratar esclerose lateral amiotrófica - Google Patents

Sequências antissenso para tratar esclerose lateral amiotrófica

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Publication number
BR112022020412A2
BR112022020412A2 BR112022020412A BR112022020412A BR112022020412A2 BR 112022020412 A2 BR112022020412 A2 BR 112022020412A2 BR 112022020412 A BR112022020412 A BR 112022020412A BR 112022020412 A BR112022020412 A BR 112022020412A BR 112022020412 A2 BR112022020412 A2 BR 112022020412A2
Authority
BR
Brazil
Prior art keywords
lateral sclerosis
amyotrophic lateral
sense sequences
treat amyotrophic
sequences
Prior art date
Application number
BR112022020412A
Other languages
English (en)
Inventor
Biferi Maria-Grazia
Cappella Marisa
Barkats Martine
Original Assignee
Association Inst De Myologie
Inst Nat Sante Rech Med
Univ Sorbonne
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Association Inst De Myologie, Inst Nat Sante Rech Med, Univ Sorbonne filed Critical Association Inst De Myologie
Publication of BR112022020412A2 publication Critical patent/BR112022020412A2/pt

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    • 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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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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Abstract

SEQUÊNCIAS ANTISSENSO PARA TRATAR ESCLEROSE LATERAL AMIOTRÓFICA. A presente invenção se refere a sequências antissenso, construtos de ácido nucleico e vetores que compreendem as ditas sequências antissenso, e seu uso para tratar uma doença associada à expansão de repetição de hexanucleotídeo C9orf72 como esclerose lateral amiotrófica ou demência frontotemporal.
BR112022020412A 2020-04-09 2021-04-09 Sequências antissenso para tratar esclerose lateral amiotrófica BR112022020412A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP20169064 2020-04-09
PCT/EP2021/059313 WO2021205005A2 (en) 2020-04-09 2021-04-09 Antisense sequences for treating amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
BR112022020412A2 true BR112022020412A2 (pt) 2023-05-02

Family

ID=70285522

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022020412A BR112022020412A2 (pt) 2020-04-09 2021-04-09 Sequências antissenso para tratar esclerose lateral amiotrófica

Country Status (11)

Country Link
US (1) US20230139408A1 (pt)
EP (1) EP4133073A2 (pt)
JP (1) JP2023520730A (pt)
KR (1) KR20230009383A (pt)
CN (1) CN115516093A (pt)
AU (1) AU2021253183A1 (pt)
BR (1) BR112022020412A2 (pt)
CA (1) CA3171436A1 (pt)
IL (1) IL296702A (pt)
MX (1) MX2022012637A (pt)
WO (1) WO2021205005A2 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023183304A2 (en) * 2022-03-21 2023-09-28 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
ES2566553T3 (es) 2010-03-17 2016-04-13 Association Institut De Myologie ARNnp U7 modificados para el tratamiento de las enfermedades neuromusculares
DK2581448T3 (en) 2011-10-13 2015-04-27 Ass Inst De Myologie Tricyclo-DNA phosphorothioate
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie 遺伝子治療ベクターの広範な遺伝子送達
WO2014062736A1 (en) * 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
WO2015054676A2 (en) * 2013-10-11 2015-04-16 Isis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2015057727A1 (en) * 2013-10-14 2015-04-23 Isis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016024205A1 (en) * 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
WO2016112132A1 (en) * 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10407678B2 (en) * 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
SG10202102997UA (en) * 2016-09-30 2021-04-29 Regeneron Pharma Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus
US11739325B2 (en) * 2017-08-08 2023-08-29 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
US10905707B2 (en) * 2017-11-10 2021-02-02 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders

Also Published As

Publication number Publication date
IL296702A (en) 2022-11-01
US20230139408A1 (en) 2023-05-04
MX2022012637A (es) 2022-11-07
WO2021205005A2 (en) 2021-10-14
CN115516093A (zh) 2022-12-23
WO2021205005A3 (en) 2021-11-18
JP2023520730A (ja) 2023-05-18
CA3171436A1 (en) 2021-10-14
AU2021253183A1 (en) 2022-11-03
KR20230009383A (ko) 2023-01-17
EP4133073A2 (en) 2023-02-15

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