BR112021024828A2 - Construtos de edição de gene alvejado e métodos de uso dos mesmos - Google Patents

Construtos de edição de gene alvejado e métodos de uso dos mesmos

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Publication number
BR112021024828A2
BR112021024828A2 BR112021024828A BR112021024828A BR112021024828A2 BR 112021024828 A2 BR112021024828 A2 BR 112021024828A2 BR 112021024828 A BR112021024828 A BR 112021024828A BR 112021024828 A BR112021024828 A BR 112021024828A BR 112021024828 A2 BR112021024828 A2 BR 112021024828A2
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Brazil
Prior art keywords
nucleic acid
methods
encodes
gene editing
targeted gene
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BR112021024828A
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English (en)
Inventor
Sánchez-Mejías García Avencia
Ivancic Djermanovic Dimitrie
Güell Cargol Marc
Pallarès Masmitja Maria
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Univ Pompeu Fabra
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Publication date
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Publication of BR112021024828A2 publication Critical patent/BR112021024828A2/pt

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N15/113—Non-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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Abstract

construtos de edição de gene alvejado e métodos de uso dos mesmos. a presente invenção fornece construtos de ácido nucleico para uso no aprimoramento da inserção sítio específica de um ácido nucleico exógeno em um genoma. em alguns aspectos, o construto de ácido nucleico compreendendo uma primeira sequência polinucleotídica que codifica uma proteína de ligação a dna manipulada para se ligar a uma sequência de dna genômico específica, um segundo polinucleotídeo compreendendo uma integrase modificada ou uma transposase modificada que permite a inserção de ácido nucleico exógeno no genoma e uma sequência de ácidos nucleicos que codifica um ligante entre os dois nucleotídeos. em algumas modalidades, o construto de ácido nucleico codifica uma proteína de fusão, por exemplo, uma proteína de fusão para entrega para uma célula por uma partícula lentiviral.
BR112021024828A 2019-06-11 2020-06-11 Construtos de edição de gene alvejado e métodos de uso dos mesmos BR112021024828A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962860186P 2019-06-11 2019-06-11
PCT/IB2020/055507 WO2020250181A1 (en) 2019-06-11 2020-06-11 Targeted gene editing constructs and methods of using the same

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BR112021024828A2 true BR112021024828A2 (pt) 2022-01-25

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US (1) US20220235379A1 (pt)
EP (1) EP3983541A1 (pt)
JP (1) JP2022540318A (pt)
KR (1) KR20220019794A (pt)
CN (1) CN114026240A (pt)
AU (1) AU2020290790A1 (pt)
BR (1) BR112021024828A2 (pt)
CA (1) CA3141422A1 (pt)
IL (1) IL288794A (pt)
MX (1) MX2021015157A (pt)
WO (1) WO2020250181A1 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022129438A1 (en) 2020-12-16 2022-06-23 Universitat Pompeu Fabra Programmable transposases and uses thereof
AU2021399929A1 (en) * 2020-12-16 2023-07-06 Universitat Pompeu Fabra Therapeutic lama2 payload for treatment of congenital muscular dystrophy
JP2024518094A (ja) * 2021-05-14 2024-04-24 ベクトン・ディキンソン・アンド・カンパニー 多重非バイアス核酸増幅法
IL311782A (en) * 2021-10-04 2024-05-01 Poseida Therapeutics Inc Transposases and their uses
WO2023129940A1 (en) * 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof
WO2023141504A2 (en) * 2022-01-19 2023-07-27 Genomeminer, Inc. (Formally Tupac Bio, Inc.) Dcas9-integrase for targeted genome editing

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI1546322T1 (sl) * 2002-07-24 2011-05-31 Manoa Biosciences Inc Vektorji na osnovi transpozona in metode integracije nukleinskih kislin
EP3277805A1 (en) * 2015-03-31 2018-02-07 Exeligen Scientific, Inc. Cas 9 retroviral integrase and cas 9 recombinase systems for targeted incorporation of a dna sequence into a genome of a cell or organism
US20200377881A1 (en) * 2017-03-24 2020-12-03 President And Fellows Of Harvard College Methods of Genome Engineering by Nuclease-Transposase Fusion Proteins

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AU2020290790A1 (en) 2022-01-27
KR20220019794A (ko) 2022-02-17
MX2021015157A (es) 2022-03-17
WO2020250181A1 (en) 2020-12-17
EP3983541A1 (en) 2022-04-20
IL288794A (en) 2022-02-01
CN114026240A (zh) 2022-02-08
US20220235379A1 (en) 2022-07-28
JP2022540318A (ja) 2022-09-15
CA3141422A1 (en) 2020-12-17

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