JP5864100B2 - 組織特異的ペプチドコンジュゲートおよび方法 - Google Patents

組織特異的ペプチドコンジュゲートおよび方法 Download PDF

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JP5864100B2
JP5864100B2 JP2010514854A JP2010514854A JP5864100B2 JP 5864100 B2 JP5864100 B2 JP 5864100B2 JP 2010514854 A JP2010514854 A JP 2010514854A JP 2010514854 A JP2010514854 A JP 2010514854A JP 5864100 B2 JP5864100 B2 JP 5864100B2
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JP2010532168A5 (enExample
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ホン エム． モールトン
ホン エム． モールトン
パトリック エル． アイバーセン
パトリック エル． アイバーセン
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サレプタ セラピューティクス インコーポレイテッド
サレプタ セラピューティクス インコーポレイテッド
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
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    • A61P35/00—Antineoplastic agents
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

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JP2010514854A 2007-06-29 2008-06-30 組織特異的ペプチドコンジュゲートおよび方法 Active JP5864100B2 (ja)

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US93772507P 2007-06-29 2007-06-29
US60/937,725 2007-06-29
PCT/US2008/008168 WO2009005793A2 (en) 2007-06-29 2008-06-30 Tissue specific peptide conjugates and methods

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JP2015182739A Active JP6584888B2 (ja) 2007-06-29 2015-09-16 組織特異的ペプチドコンジュゲートおよび方法
JP2018121949A Pending JP2018171066A (ja) 2007-06-29 2018-06-27 組織特異的ペプチドコンジュゲートおよび方法

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US (1) US20090099066A1 (enExample)
EP (2) EP3443976A1 (enExample)
JP (3) JP5864100B2 (enExample)
AU (1) AU2008271050B2 (enExample)
CA (1) CA2691673A1 (enExample)
ES (1) ES2694726T3 (enExample)
WO (1) WO2009005793A2 (enExample)

Families Citing this family (117)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003225410A1 (en) * 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP2351844B1 (en) 2003-04-29 2014-06-11 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
WO2005072527A2 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
HRP20110352T1 (hr) 2004-06-28 2011-06-30 The University Of Western Australia Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
CA2596506C (en) * 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8168181B2 (en) 2006-02-13 2012-05-01 Alethia Biotherapeutics, Inc. Methods of impairing osteoclast differentiation using antibodies that bind siglec-15
EP2438962A3 (en) 2006-02-13 2012-07-18 Alethia Biotherapeutics Inc. Polynucleotides and polypeptide sequences involved in the process of bone remodeling
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2704049A1 (en) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US7989608B2 (en) * 2007-12-28 2011-08-02 Avi Biopharma Inc. Immunomodulatory agents and methods of use
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8231895B2 (en) 2008-05-22 2012-07-31 Universidade De Coimbra Targeted delivery to human diseases and disorders
WO2009144481A2 (en) * 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
JP2011523557A (ja) * 2008-06-04 2011-08-18 メディカル リサーチ カウンシル ペプチド
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
US8592386B2 (en) * 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
KR102113306B1 (ko) 2009-11-12 2020-05-21 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
KR101944119B1 (ko) 2009-11-13 2019-01-30 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 항바이러스 화합물 및 인플루엔자 바이러스 감염을 치료하는 방법
KR20120125455A (ko) * 2009-12-11 2012-11-15 광주과학기술원 세포내 타겟 결합용 바이포달 펩타이드 바인더
WO2011127210A1 (en) * 2010-04-06 2011-10-13 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
KR102095478B1 (ko) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
JP5981428B2 (ja) * 2010-07-19 2016-08-31 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
CA2834128A1 (en) * 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
EP2751128B1 (en) 2011-08-30 2021-03-03 United Kingdom Research and Innovation Cell-penetrating peptides having a central hydrophobic domain
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
PT2581448E (pt) * 2011-10-13 2015-05-21 Institut National De La Santé Et De La Rech Médicale (Inserm) Dna triciclo-fosforotioato
SI2788487T1 (sl) 2011-12-08 2018-09-28 Sarepta Therapeutics, Inc. Analogi oligonukleotida, ki ciljajo humano LMNA
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US10059941B2 (en) * 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
SG11201407486PA (en) * 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating utrn expression
US10174323B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating ATP2A2 expression
CN104583399A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节血红蛋白基因家族表达的组合物和方法
KR101329411B1 (ko) 2012-05-31 2013-11-14 주식회사 엘지생활건강 피부투과성 펩타이드
CA2928851A1 (en) 2012-07-19 2014-01-23 Alethia Biotherapeutics Inc. Anti-siglec-15 antibodies
ES2941477T3 (es) 2012-08-13 2023-05-23 Univ Rockefeller Agonista de LXRbeta para el tratamiento de cáncer
MX369519B (es) 2012-09-25 2019-11-11 Genzyme Corp Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.
NZ708171A (en) 2012-11-15 2019-11-29 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
CN103981147B (zh) 2013-02-08 2017-11-10 中国科学院上海生命科学研究院 一种新的制备肝实质细胞的方法
NZ731587A (en) 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
CN113633787A (zh) 2013-03-15 2021-11-12 萨勒普塔医疗公司 改进的用于治疗肌营养不良的组合物
WO2015069586A2 (en) 2013-11-06 2015-05-14 Merck Sharp & Dohme Corp. Dual molecular delivery of oligonucleotides and peptide containing conjugates
US9790495B2 (en) 2014-05-16 2017-10-17 Oregon State University Antisense antibacterial compounds and methods
US10391098B2 (en) 2014-05-19 2019-08-27 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
TWI713450B (zh) 2014-05-23 2020-12-21 美商健臻公司 藉由使用反義寡核苷酸創造提前終止密碼子抑制或下調肝醣合成酶
KR102487942B1 (ko) 2014-05-23 2023-01-11 젠자임 코포레이션 펩티드 담체 상의 다중 올리고뉴클레오티드 모이어티
AU2015372560B2 (en) * 2014-12-31 2021-12-02 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
MA41795A (fr) * 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
BR112018007066A2 (pt) * 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
CA3009342A1 (en) 2015-12-23 2017-06-29 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
US10907158B2 (en) 2015-12-23 2021-02-02 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
SG11201809468XA (en) 2016-04-29 2018-11-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
HRP20221316T1 (hr) * 2016-12-19 2022-12-23 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
PL3554553T3 (pl) 2016-12-19 2022-11-07 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
ES2928486T3 (es) 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Conjugados de oligómeros con omisión de exón para la distrofia muscular
US10800817B2 (en) * 2016-12-19 2020-10-13 Morehouse School Of Medicine Compositions and methods for treating diseases by inhibiting exosome release
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US20180207293A1 (en) * 2017-01-25 2018-07-26 2C Tech Corp. Nanoparticles for sustained ophthalmic drug delivery and methods of use
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
JP2020536058A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
EP3687547A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP2020536057A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
WO2019079386A1 (en) * 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION
AU2018373028A1 (en) 2017-11-21 2020-04-30 Inspirna, Inc. Polymorphs and uses thereof
JP7013626B2 (ja) * 2018-01-05 2022-02-01 国立医薬品食品衛生研究所長 細胞膜透過ペプチド、構築物、及び、カーゴ分子を細胞内に輸送する方法
EP3790890A4 (en) 2018-05-09 2022-03-02 Ohio State Innovation Foundation CYCLIC, CELL-PENETRATING PEPTIDES WITH ONE OR MORE HYDROPHOBIC RAILS
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR20210081323A (ko) * 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 근긴장성 이영양증을 치료하기 위한 그의 용도
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
CN113453723A (zh) 2018-12-07 2021-09-28 牛津大学科技创新有限公司 接头
AU2019397461A1 (en) 2018-12-13 2021-07-29 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof
KR102270700B1 (ko) * 2019-11-01 2021-06-30 한국과학기술연구원 폐 섬유증의 예방 또는 치료를 위한 올리고뉴클레오티드 중합체와 생체 적합성 양이온성 펩타이드로 이루어지는 폐 특이적 약물 전달체 및 이의 이용
HUE067466T2 (hu) 2019-12-13 2024-10-28 Inspirna Inc Fémsók és alkalmazásuk
CN114901823A (zh) 2019-12-26 2022-08-12 日本新药株式会社 诱导外显子50的跳读的反义核酸
JPWO2021172498A1 (enExample) 2020-02-28 2021-09-02
US11987795B2 (en) 2020-11-24 2024-05-21 The Broad Institute, Inc. Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11
US20240327831A1 (en) 2020-12-23 2024-10-03 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
EP4330395A1 (en) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
AU2022298028A1 (en) 2021-06-23 2023-12-21 National Center Of Neurology And Psychiatry Combination of antisense oligomers
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
US20240285770A1 (en) 2021-07-08 2024-08-29 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4387676A2 (en) * 2021-09-03 2024-06-26 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
AU2023235302A1 (en) 2022-03-17 2024-10-24 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
CN119183457A (zh) 2022-04-15 2024-12-24 达因疗法公司 用于治疗强直性肌营养不良的肌肉靶向复合物和制剂
US20250289851A1 (en) 2022-04-22 2025-09-18 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
WO2024091824A1 (en) 2022-10-26 2024-05-02 Ada Forsyth Institute, Inc. Differentiation and reprogramming of chondrocyte
EP4612292A1 (en) 2022-11-02 2025-09-10 Sarepta Therapeutics, Inc. Formulation of an antisense oligomer conjugate
WO2024129459A1 (en) 2022-12-16 2024-06-20 University Of Rochester Repairmen! of barrier dysfunction in esophagus
WO2025085868A2 (en) 2023-10-20 2025-04-24 Radiation Control Technologies, Inc. Cd47-targeting morpholinos

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US946496A (en) 1909-08-21 1910-01-11 Henry J Cox Fulcrum-block.
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
DE3650699T2 (de) 1985-03-15 1999-04-15 Antivirals Inc., Portland, Oreg. Immunotestmittel für Polynukleotid und Verfahren
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5525465A (en) * 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
US5747641A (en) * 1989-12-21 1998-05-05 Biogen Inc Tat-derived transport polypeptide conjugates
DK0690726T3 (da) * 1993-01-07 2002-03-11 Univ Jefferson Antisense-inhibibering af c-myc til modulering af proliferationen af glat muskel-celler
US6686338B1 (en) 1996-02-23 2004-02-03 The Board Of Regents Of The University Of Nebraska Enzyme inhibitors for metabolic redirection
US5849727A (en) * 1996-06-28 1998-12-15 Board Of Regents Of The University Of Nebraska Compositions and methods for altering the biodistribution of biological agents
ES2210761T3 (es) * 1997-05-21 2004-07-01 The Board Of Trustees Of The Leland Stanford Junior University Composicion y procedimiento para mejorar el transporte a traves de las membranas biologicas.
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
AUPP398498A0 (en) 1998-06-09 1998-07-02 Silverbrook Research Pty Ltd A method of manufacture of an image creation apparatus (ijm44)
JP2002520298A (ja) 1998-07-13 2002-07-09 ザ・ボード・オブ・リージェンツ・オブ・ザ・ユニバーシティー・オブ・ネブラスカ 標的化部位特異的薬物送達組成物および使用方法
JP2002535015A (ja) 1999-01-29 2002-10-22 エイブイアイ バイオファーマ， インコーポレイテッド 標的ｒｎａを検出するための非侵襲性方法
US7094765B1 (en) 1999-01-29 2006-08-22 Avi Biopharma, Inc. Antisense restenosis composition and method
EP1173614A4 (en) * 1999-04-08 2003-10-29 Oasis Biosciences Inc ANTISENSE OLIGONUCLEOTIDS CONTAINING UNIVERSAL AND / OR DEGENERATED BASES
US6303573B1 (en) * 1999-06-07 2001-10-16 The Burnham Institute Heart homing peptides and methods of using same
US6669951B2 (en) * 1999-08-24 2003-12-30 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into epithelial tissues
AU2001263197B2 (en) 2000-05-17 2006-11-09 The Board Of Regents Of The University Of Nebraska Antisense enzyme inhibitors for metabolic redirection
US20040170955A1 (en) 2000-09-08 2004-09-02 Wadih Arap Human and mouse targeting peptides identified by phage display
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
WO2002063280A1 (en) * 2001-02-06 2002-08-15 Auburn University Ligand sensor devices and uses thereof
WO2002065986A2 (en) * 2001-02-16 2002-08-29 Cellgate, Inc. Transporters comprising spaced arginine moieties
JP2004537517A (ja) * 2001-05-17 2004-12-16 エイブイアイ バイオファーマ， インコーポレイテッド ｃ−ｍｙｃアンチセンスオリゴマーを使用した、癌を処置するための併用アプローチ
JP3735292B2 (ja) * 2001-07-26 2006-01-18 三菱重工業株式会社 ダイエット効果のある健康食品および製剤
US6645974B2 (en) * 2001-07-31 2003-11-11 Merck & Co., Inc. Androgen receptor modulators and methods for use thereof
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
CA2469336C (en) * 2001-12-11 2013-06-11 The Board Of Trustees Of The Leland Stanford Junior University Guanidinium transport reagents and conjugates
US20030207907A1 (en) 2002-05-03 2003-11-06 Iversen Patrick L. Delivery of microparticle-conjugated drugs for inhibition of stenosis
EP2351844B1 (en) * 2003-04-29 2014-06-11 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US20050203041A1 (en) 2003-09-23 2005-09-15 Mourich Dan V. Antisense compound and method for selectively killing activated T cells
US7786151B2 (en) * 2004-01-09 2010-08-31 Kinopharma, Inc. Therapeutic composition of treating abnormal splicing caused by the excessive kinase induction
WO2005072527A2 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20060078542A1 (en) * 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
WO2005117992A2 (en) * 2004-05-30 2005-12-15 Cemines, Inc. Controlled delivery of therapeutic compounds
HRP20110352T1 (hr) 2004-06-28 2011-06-30 The University Of Western Australia Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe
US7489981B2 (en) * 2004-09-01 2009-02-10 Honda Motor Co., Ltd. Assembly line control system and immobilizer data protocol and communication process flow
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8357664B2 (en) * 2004-10-26 2013-01-22 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
NZ538097A (en) * 2005-02-07 2006-07-28 Ovita Ltd Method and compositions for improving wound healing
CA2596506C (en) * 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US7790694B2 (en) * 2005-07-13 2010-09-07 Avi Biopharma Inc. Antisense antibacterial method and compound
WO2007030576A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8501704B2 (en) * 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
CA2628093A1 (en) * 2005-11-08 2007-05-18 Avi Biopharma, Inc. Antisense oligonucleotide analog compounds targeting proprocessed ctla-4 mrna
EP2002018B1 (en) * 2006-03-07 2010-05-12 AVI BioPharm, Inc. Antisense antiviral compound and method for treating arenavirus infection
DK2024499T3 (da) 2006-05-10 2018-01-29 Sarepta Therapeutics Inc Oligonukleotidanaloger med kationiske intersubunit-koblinger
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US7989608B2 (en) * 2007-12-28 2011-08-02 Avi Biopharma Inc. Immunomodulatory agents and methods of use
WO2010048586A1 (en) * 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
US8592386B2 (en) * 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis

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