BR112021007403A2 - Composições e métodos para entrega de transgenes - Google Patents

Composições e métodos para entrega de transgenes

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Publication number
BR112021007403A2
BR112021007403A2 BR112021007403A BR112021007403A BR112021007403A2 BR 112021007403 A2 BR112021007403 A2 BR 112021007403A2 BR 112021007403 A BR112021007403 A BR 112021007403A BR 112021007403 A BR112021007403 A BR 112021007403A BR 112021007403 A2 BR112021007403 A2 BR 112021007403A2
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BR
Brazil
Prior art keywords
compositions
methods
systems
transgene
transgene delivery
Prior art date
Application number
BR112021007403A
Other languages
English (en)
Inventor
Richard Brooks Alan
Karen Vo
Original Assignee
Bayer Healthcare Llc
Crispr Therapeutics Ag
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 Bayer Healthcare Llc, Crispr Therapeutics Ag filed Critical Bayer Healthcare Llc
Publication of BR112021007403A2 publication Critical patent/BR112021007403A2/pt

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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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    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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Abstract

composições e métodos para entrega de transgenes. trata-se de composições, métodos e sistemas para distribuição direcionada de ácidos nucleicos, incluindo dna e rna, para uma célula alvo. também são fornecidos composições, métodos e sistemas para expressar um transgene em uma célula por edição genômica. além disso, são fornecidas composições, métodos e sistemas para bater em um gene de interesse (goi) em um locus genômico alvo no genoma, em particular o locus do gene da albumina. também são fornecidos composições, métodos e sistemas para o tratamento de um sujeito com ou suspeito de ter um distúrbio ou condição de saúde empregando edição do genoma ex vivo e / ou in vivo.
BR112021007403A 2018-10-17 2019-10-17 Composições e métodos para entrega de transgenes BR112021007403A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862747128P 2018-10-17 2018-10-17
PCT/US2019/056779 WO2020081843A1 (en) 2018-10-17 2019-10-17 Compositions and methods for delivering transgenes

Publications (1)

Publication Number Publication Date
BR112021007403A2 true BR112021007403A2 (pt) 2021-12-07

Family

ID=68468827

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021007403A BR112021007403A2 (pt) 2018-10-17 2019-10-17 Composições e métodos para entrega de transgenes

Country Status (11)

Country Link
US (1) US20210348159A1 (pt)
EP (1) EP3867377A1 (pt)
JP (1) JP7520826B2 (pt)
KR (1) KR20210096088A (pt)
CN (1) CN113366106A (pt)
AU (1) AU2019362000A1 (pt)
BR (1) BR112021007403A2 (pt)
CA (1) CA3116885A1 (pt)
IL (1) IL282369A (pt)
MX (1) MX2021004455A (pt)
WO (1) WO2020081843A1 (pt)

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CN113939595A (zh) 2019-06-07 2022-01-14 瑞泽恩制药公司 包括人源化白蛋白基因座的非人动物
IL302335A (en) * 2020-10-26 2023-06-01 Res Inst Nationwide Childrens Hospital NK cells with chimeric antigen receptor (CAR) and uses thereof
CN114496063B (zh) * 2022-01-13 2023-05-23 北京博康健基因科技有限公司 基于天然氨基酸序列的信号肽设计与二级结构从头算建模方法和装置
WO2024006960A1 (en) * 2022-06-29 2024-01-04 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids
CN117947040A (zh) * 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用
WO2024157194A1 (en) * 2023-01-25 2024-08-02 Crispr Therapeutics Ag Methods and assays for off-target analysis

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