BR0014814A - Método para alterar a expressão de uma seq ência alvo de ácido nucléico endógeno em uma célula alvo, transcrição de cdna e célula alvo - Google Patents

Método para alterar a expressão de uma seq ência alvo de ácido nucléico endógeno em uma célula alvo, transcrição de cdna e célula alvo

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Publication number
BR0014814A
BR0014814A BR0014814-8A BR0014814A BR0014814A BR 0014814 A BR0014814 A BR 0014814A BR 0014814 A BR0014814 A BR 0014814A BR 0014814 A BR0014814 A BR 0014814A
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Brazil
Prior art keywords
sequence
target cell
cdna
target
expression
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BR0014814-8A
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English (en)
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Charles A Conrad
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Cytogenix Inc
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Publication date
Application filed by Cytogenix Inc filed Critical Cytogenix Inc
Publication of BR0014814A publication Critical patent/BR0014814A/pt

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

"MéTODO PARA ALTERAR A EXPRESSãO DE UMA SEQ êNCIA ALVO DE áCIDO NUCLéICO ENDóGENO EM UMA CéLULA ALVO, TRANSCRIçãO DE CDNA E CéLULA ALVO". A presente invenção se refere a métodos para alterar a expressão de uma sequência alvo de ácido nucléico em uma célula alvo pela produção de cDNA(ss-cDNA) de cordão único na célula alvo in vivo. A célula alvo é transfectada com um cassete compreendendo uma seq³ência de interesse, uma repetição conjugada invertida e um local de ligação prímero 3' (PBS) para a repetição conjugada invertida. A transcrição do cassete pela célula alvo produz um templato RNA o qual é reversamente transcrito para produzir ss-cDNA de uma seq³ência específica. O gene de codificação transcriptase reversa/RNase também pode ser transfectado para a interior da célula de alvo. O ss-cDNA é modificado para remover todos os vetores de flanqueamento pelo tirar proveito da estrutura de enlace do ss-cDNA, a qual se forma como um resultado da repetição conjugada invertida que permite ao ss-cDNA dobrar-se sobre si mesmo, formando um cadeia de DNA de cordão duplo. A cadeia de cordão duplo contem um ou mais locais de restrição de reconhecimento de endonuclease e o enlace, o qual permanece como ssDNA, é compreendido da seq³ência de interesse, a qual pode ser qualquer seq³ência de nucleotídeos desejada. Este desenho permite à cadeia de cordão duplo do enlace de cadeia intermediário ser clivada pela(s) restrição(ões) de endonuclease correspondente(s) desejada(s) e a porção de enlace da seq³ência de interesse é então liberada como uma peça de DNA de cordão único, linearizada. Esta peça de ssDNA liberada (ou clivada) contem uma informação de seq³ência mínima, se ainda houver, seja a montante de 5' ou à jusante de 3' a partir da cadeia de cordão duplo. O ssDNA resultante se liga a uma seq³ência alvo de ácido nucléico endógeno para alterar a expressão daquela seq³ência para finalidades terapêuticas tais como a desativação de um gene usando uma ligação dupla ou tripla de ácidos nucléicos mutagenesis direcionados por local, a interrupção da função celular pela ligação a proteínas celulares específicas, e interferir com as funções de divisão de RNA.
BR0014814-8A 1999-10-04 2000-10-04 Método para alterar a expressão de uma seq ência alvo de ácido nucléico endógeno em uma célula alvo, transcrição de cdna e célula alvo BR0014814A (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41156899A 1999-10-04 1999-10-04
US51470700A 2000-02-28 2000-02-28
PCT/US2000/027381 WO2001025419A1 (en) 1999-10-04 2000-10-04 ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO

Publications (1)

Publication Number Publication Date
BR0014814A true BR0014814A (pt) 2002-09-17

Family

ID=27021437

Family Applications (1)

Application Number Title Priority Date Filing Date
BR0014814-8A BR0014814A (pt) 1999-10-04 2000-10-04 Método para alterar a expressão de uma seq ência alvo de ácido nucléico endógeno em uma célula alvo, transcrição de cdna e célula alvo

Country Status (11)

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EP (1) EP1222259A1 (pt)
JP (1) JP2003511025A (pt)
KR (1) KR20020059608A (pt)
CN (1) CN1276083C (pt)
AU (1) AU7855300A (pt)
BR (1) BR0014814A (pt)
CA (1) CA2386246A1 (pt)
CR (1) CR6613A (pt)
IL (1) IL148946A0 (pt)
MX (1) MXPA02003422A (pt)
WO (1) WO2001025419A1 (pt)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7419964B2 (en) * 1999-09-16 2008-09-02 Cytogenix, Inc. Treatment of HSV-related pathologies using ssDNA
EP1399585B1 (en) 2000-10-23 2008-02-20 Gen-Probe Incorporated Compositions and methods for detecting human immunodeficiency virus 2 (hiv-2)
IL162462A0 (en) * 2001-12-14 2005-11-20 Univ Yale Intracellular generation of single-stranded dna
US20050020526A1 (en) * 2003-06-03 2005-01-27 Cytogenix, Inc. Oligodeoxynucleotide intervention for prevention and treatment of sepsis
EP1802754A2 (en) * 2004-09-28 2007-07-04 CytoGenix, Inc. Single-stranded antimicrobial oligonucleotides and uses thereof
US8658608B2 (en) 2005-11-23 2014-02-25 Yale University Modified triple-helix forming oligonucleotides for targeted mutagenesis
CN102154269B (zh) * 2011-01-06 2012-05-23 湖南农业大学 基于pcr的串联重复序列快速构建法和专用引物对及应用
JP2019508037A (ja) 2016-02-16 2019-03-28 イェール ユニバーシティーＹａｌｅ Ｕｎｉｖｅｒｓｉｔ 標的化遺伝子編集を増強するための組成物およびその使用方法
CN108396043A (zh) * 2017-02-06 2018-08-14 中国科学院上海应用物理研究所 一种5`端磷酸化单链dna的制备方法及其应用
WO2018187493A1 (en) 2017-04-04 2018-10-11 Yale University Compositions and methods for in utero delivery
JP7481755B2 (ja) * 2018-11-02 2024-05-13 ナイキジェン，リミテッド 組換えパルボウイルスベクターならびにその作製および使用方法
GB201913898D0 (en) * 2019-09-26 2019-11-13 Lightbio Ltd Nucleic acid construct
CN116286979A (zh) * 2023-03-07 2023-06-23 长沙忠健科技有限公司 一种表达逆转录酶和rna序列的质粒、细胞及其应用

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MXPA01003643A (es) * 1998-10-09 2003-07-21 Ingene Inc Produccion de adnss in vivo.
JP2002527061A (ja) * 1998-10-09 2002-08-27 インジーン・インコーポレイテッド ｓｓＤＮＡの酵素的合成

Also Published As

Publication number Publication date
CN1276083C (zh) 2006-09-20
CA2386246A1 (en) 2001-04-12
KR20020059608A (ko) 2002-07-13
JP2003511025A (ja) 2003-03-25
AU7855300A (en) 2001-05-10
WO2001025419A1 (en) 2001-04-12
EP1222259A1 (en) 2002-07-17
MXPA02003422A (es) 2004-09-10
CN1399679A (zh) 2003-02-26
CR6613A (es) 2004-04-21
IL148946A0 (en) 2002-09-12

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