OA10194A - Ribozyme gene therapy for hiv infection and aids - Google Patents

Ribozyme gene therapy for hiv infection and aids Download PDF

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Publication number
OA10194A
OA10194A OA60740A OA60740A OA10194A OA 10194 A OA10194 A OA 10194A OA 60740 A OA60740 A OA 60740A OA 60740 A OA60740 A OA 60740A OA 10194 A OA10194 A OA 10194A
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OA
OAPI
Prior art keywords
hiv
cell
cells
ribozyme
human
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OA60740A
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English (en)
Inventor
Flossie Wong-Staal
Mang Yu
Osamu Yamada
Joshua Ojwang
Mark Leavitt
Anthony Ho
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Univ California
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Publication of OA10194A publication Critical patent/OA10194A/en

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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
    • C12N15/11—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
    • C12N15/1131—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 against viruses
    • C12N15/1132—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 against viruses against retroviridae, e.g. HIV
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C—CHEMISTRY; METALLURGY
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • AIDS & HIV (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Oncology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
OA60740A 1993-05-17 1995-11-16 Ribozyme gene therapy for hiv infection and aids OA10194A (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US6246593A 1993-05-17 1993-05-17

Publications (1)

Publication Number Publication Date
OA10194A true OA10194A (en) 1996-12-18

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ID=22042666

Family Applications (1)

Application Number Title Priority Date Filing Date
OA60740A OA10194A (en) 1993-05-17 1995-11-16 Ribozyme gene therapy for hiv infection and aids

Country Status (12)

Country Link
US (3) US5670361A (no)
EP (1) EP0702716A4 (no)
JP (1) JPH08510134A (no)
KR (1) KR960702518A (no)
CN (1) CN1127527A (no)
AU (1) AU680921B2 (no)
CA (1) CA2163129A1 (no)
FI (1) FI955509A (no)
NO (1) NO954635L (no)
NZ (1) NZ267797A (no)
OA (1) OA10194A (no)
WO (1) WO1994026877A1 (no)

Families Citing this family (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU1124997A (en) * 1995-11-28 1998-06-19 Johns Hopkins University School Of Medicine, The Conditionally replicating viral vectors and their use
US5885806A (en) * 1995-11-28 1999-03-23 The Johns Hopkins University School Of Medicine Methods to prepare conditionally replicating viral vectors
US5888767A (en) 1996-11-27 1999-03-30 The Johns Hopkins University School Of Medicine Method of using a conditionally replicating viral vector to express a gene
US6426412B1 (en) 1995-12-20 2002-07-30 Subsidiary No. 3, Inc. Nucleic acids encoding human immunodeficiency virus type 1 genetic suppressor elements
US6316210B1 (en) 1995-12-20 2001-11-13 Subsidiary No. 3, Inc. Genetic suppressor elements against human immunodeficiency virus
US6200811B1 (en) * 1996-04-02 2001-03-13 The Regents Of The University Of California Cell transformation vector comprising an HIV-2 packaging site nucleic acid and an HIV-1 GAG protein
US6776986B1 (en) 1996-06-06 2004-08-17 Novartis Ag Inhibition of HIV-1 replication by antisense RNA expression
US5741706A (en) * 1996-06-13 1998-04-21 Immusol, Incorporated Anti-HIV ribozymes
US5856462A (en) * 1996-09-10 1999-01-05 Hybridon Incorporated Oligonucleotides having modified CpG dinucleosides
US6605429B1 (en) 1997-01-23 2003-08-12 Immusol, Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries
US6071743A (en) 1997-06-02 2000-06-06 Subsidiary No. 3, Inc. Compositions and methods for inhibiting human immunodeficiency virus infection by down-regulating human cellular genes
US6537972B1 (en) 1997-06-02 2003-03-25 Subsidiary No. 3., Inc. Compositions and methods for inhibiting human immunodeficiency virus infection by down-regulating human cellular genes
EP1017797B1 (en) 1997-09-24 2005-06-22 The Regents Of The University Of California Non-primate lentiviral vectors and packaging systems
ATE344322T1 (de) 1998-02-13 2006-11-15 Koester Hubert Verwendung von ribozymen zur bestimmung der funktion von genen
US6613506B1 (en) 2000-11-28 2003-09-02 Subsidiary No. 3, Inc. Compositions and methods for inhibiting human immunodeficiency virus infection by down-regulating human cellular genes
JP2990268B1 (ja) 1998-08-31 1999-12-13 工業技術院長 機能性核酸転写用発現系
US6939712B1 (en) * 1998-12-29 2005-09-06 Impedagen, Llc Muting gene activity using a transgenic nucleic acid
DE19956568A1 (de) * 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US20050020524A1 (en) * 1999-04-15 2005-01-27 Monash University Hematopoietic stem cell gene therapy
US20040259803A1 (en) * 1999-04-15 2004-12-23 Monash University Disease prevention by reactivation of the thymus
US20040258672A1 (en) * 1999-04-15 2004-12-23 Monash University Graft acceptance through manipulation of thymic regeneration
US20070274946A1 (en) * 1999-04-15 2007-11-29 Norwood Immunoloty, Ltd. Tolerance to Graft Prior to Thymic Reactivation
US20040241842A1 (en) * 1999-04-15 2004-12-02 Monash University Stimulation of thymus for vaccination development
US20040265285A1 (en) * 1999-04-15 2004-12-30 Monash University Normalization of defective T cell responsiveness through manipulation of thymic regeneration
AUPR074500A0 (en) * 2000-10-13 2000-11-09 Monash University Treatment of t cell disorders
US20040138168A1 (en) * 1999-04-21 2004-07-15 Wyeth Methods and compositions for inhibiting the function of polynucleotide sequences
CN1375004A (zh) * 1999-04-21 2002-10-16 惠氏公司 抑制多核苷酸序列的功能的方法和组合物
AU5773500A (en) 1999-06-28 2001-01-31 Board Of Trustees Of The University Of Illinois, The Inhibitors of memapsin 2 and use thereof
US7829693B2 (en) * 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US6808876B1 (en) 2000-05-02 2004-10-26 Immusol, Inc. Cellular regulators of infectious agents and methods of use
US20060088512A1 (en) * 2001-10-15 2006-04-27 Monash University Treatment of T cell disorders
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
GB0104948D0 (en) * 2001-02-28 2001-04-18 Novartis Res Foundation Novel methods
US20030191073A1 (en) 2001-11-07 2003-10-09 Challita-Eid Pia M. Nucleic acid and corresponding protein entitled 161P2F10B useful in treatment and detection of cancer
US6922593B2 (en) * 2001-08-06 2005-07-26 Gideon Weiss Control of items in a complex system by using fluid models and solving continuous linear programs
CA2472927A1 (en) 2002-01-10 2003-07-17 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw A novel splice variant of myd88 and uses thereof
US20040005546A1 (en) * 2002-02-28 2004-01-08 Oncolytics Biotech Inc. Use of ribozymes in the detection of adventitious agents
CN100579577C (zh) 2002-03-15 2010-01-13 退伍军人事务研发服务部 使用细胞脱唾液酸决定簇和糖缀合物将细胞靶向组织和器官的方法和组合物
JP2005521393A (ja) * 2002-03-20 2005-07-21 マサチューセッツ インスティテュート オブ テクノロジー Ｈｉｖ治療
AU2003243151A1 (en) 2002-08-16 2004-03-03 Agensys, Inc. Nucleic acid and corresponding protein entitled 251p5g2 useful in treatment and detection of cancer
JP2007524361A (ja) 2003-02-10 2007-08-30 アジェンシス， インコーポレイテッド 膀胱癌及びその他の癌の治療及び検出に有用な名称１５８ｐ１ｄ７の核酸及び対応タンパク質
JP2007525196A (ja) 2003-05-30 2007-09-06 アジェンシス， インコーポレイテッド 前立腺幹細胞抗原（ｐｓｃａ）変異体及びその部分配列
US7407776B2 (en) 2003-09-19 2008-08-05 Sangamo Biosciences, Inc. Engineered zinc finger proteins for regulation of gene expression
US20080279812A1 (en) * 2003-12-05 2008-11-13 Norwood Immunology, Ltd. Disease Prevention and Vaccination Prior to Thymic Reactivation
EP1753871B1 (en) 2004-05-28 2015-07-15 Agensys, Inc. Antibodies and related molecules that bind to psca proteins
US7850960B2 (en) 2004-12-30 2010-12-14 University Of Washington Methods for regulation of stem cells
KR20080003390A (ko) 2005-03-31 2008-01-07 어젠시스 인코포레이티드 161ｐ2ｆ10ｂ 단백질과 결합하는 항체 및 관련 분자
JP5518711B2 (ja) 2007-09-07 2014-06-11 アジェンシス，インコーポレイテッド ２４ｐ４ｃ１２タンパク質に結合する抗体および関連分子
ITMI20080508A1 (it) * 2008-03-27 2009-09-28 Istituto Nazionale Di Genetica Molecolare Cellule ematopoietiche esprimenti la proteina krtcap3 e i leganti per la proteina krtcap3
ITMI20080865A1 (it) * 2008-05-13 2009-11-14 Istituto Naz Di Genetica Molecolare Ingm Cellule ematopoietiche esprimenti la proteina susd3 e i leganti per la proteina susd3
US20100210989A1 (en) 2008-12-23 2010-08-19 Janet Lesley Macpherson Processing blood
WO2012015938A2 (en) 2010-07-27 2012-02-02 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
JP6214530B2 (ja) 2011-07-15 2017-10-18 ザ ジェネラル ホスピタル コーポレイション 転写活性化因子様エフェクターの組立て方法
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
EP3789405A1 (en) 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
AU2014361784A1 (en) 2013-12-12 2016-06-23 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
US11795435B2 (en) 2015-05-05 2023-10-24 Mesoblast International Sárl; Potency assay
MX2017014446A (es) * 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and their uses
KR20230156150A (ko) 2016-06-17 2023-11-13 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
CA3030587A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
CA3032699A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
SG11201909856XA (en) 2017-05-04 2019-11-28 Mesoblast Int Sarl Mesenchymal lineage precursor or stem cells with enhanced immunosuppression
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
BR112021013158A2 (pt) 2019-01-02 2021-09-14 Mesoblast International Sàrl Método para tratar a dor lombar
US20220079991A1 (en) 2019-01-03 2022-03-17 Mesoblast International Sárl Method for improving visual acuity
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
KR20230085929A (ko) 2020-10-15 2023-06-14 에프. 호프만-라 로슈 아게 Va rna 전사를 위한 핵산 구조체
JP2023546113A (ja) 2020-10-15 2023-11-01 エフ． ホフマン－ラ ロシュ アーゲー 同時遺伝子活性化のための核酸構築物
CN114015674B (zh) 2021-11-02 2024-08-30 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4987071A (en) * 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5037746A (en) * 1986-12-03 1991-08-06 University Patents, Inc. RNA ribozyme polymerases, and methods
US5116742A (en) * 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US5254678A (en) * 1987-12-15 1993-10-19 Gene Shears Pty. Limited Ribozymes
US5866701A (en) * 1988-09-20 1999-02-02 The Board Of Regents For Northern Illinois University Of Dekalb HIV targeted hairpin ribozymes
CA1340323C (en) * 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
US5176996A (en) * 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5168053A (en) * 1989-03-24 1992-12-01 Yale University Cleavage of targeted RNA by RNAase P
JPH04505261A (ja) * 1989-05-10 1992-09-17 スローン―ケツテリング・インステイテユート・フオー・キヤンサー・リサーチ ポルｉｉｉプロモーターの制御下の転写可能な外来ｄｎａを含む安定的に形質転換された真核細胞
US5144019A (en) * 1989-06-21 1992-09-01 City Of Hope Ribozyme cleavage of HIV-I RNA
US5149796A (en) * 1989-08-31 1992-09-22 City Of Hope Chimeric DNA-RNA catalytic sequences
US5180818A (en) * 1990-03-21 1993-01-19 The University Of Colorado Foundation, Inc. Site specific cleavage of single-stranded dna

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