ES2657737T3 - Vector dual para la inhibición del virus de la inmunodeficiencia humana - Google Patents

Vector dual para la inhibición del virus de la inmunodeficiencia humana Download PDF

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Publication number
ES2657737T3
ES2657737T3 ES15166660.9T ES15166660T ES2657737T3 ES 2657737 T3 ES2657737 T3 ES 2657737T3 ES 15166660 T ES15166660 T ES 15166660T ES 2657737 T3 ES2657737 T3 ES 2657737T3
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Prior art keywords
nucleic acid
hiv
vector
cell
inhibition
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ES15166660.9T
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Inventor
Louis Randall Breton
Irvin Chen
Dong Sung An
Geoffrey P. Symonds
Maureen Boyd
Michelle L. Millington
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CSL Behring Gene Therapy Inc
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Calimmune Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0271—Chimeric vertebrates, e.g. comprising exogenous cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • 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
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/90—Isomerases (5.)
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0337—Animal models for infectious diseases
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • 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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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2740/16011—Human Immunodeficiency Virus, HIV
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Abstract

Un metodo para producir un vector de expresion virica que, cuando esta presente en una celula, es capaz de inhibir la union del VIH con la celula y evitar la fusion del VIH en la celula, comprendiendo el metodo: insertar en el vector una primera molecula de acido nucleico que codifica un acido nucleico inhibidor capaz de regular negativamente la expresion de un correceptor de VIH, en donde el acido nucleico inhibidor es un ARN de interferencia pequeno (ARNip) o un ARN en horquilla corto (ARNhp) que tiene una region bicatenaria; e insertar en el vector una segunda molecula de acido nucleico que codifica una proteina inhibidora de fusion de VIH

Description

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Claims (1)

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ES15166660.9T 2009-07-15 2010-05-26 Vector dual para la inhibición del virus de la inmunodeficiencia humana Active ES2657737T3 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US22568709P 2009-07-15 2009-07-15
US225687P 2009-07-15
US28759909P 2009-12-17 2009-12-17
US287599P 2009-12-17

Publications (1)

Publication Number Publication Date
ES2657737T3 true ES2657737T3 (es) 2018-03-06

Family

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Family Applications (3)

Application Number Title Priority Date Filing Date
ES15166660.9T Active ES2657737T3 (es) 2009-07-15 2010-05-26 Vector dual para la inhibición del virus de la inmunodeficiencia humana
ES10720980.1T Active ES2546663T3 (es) 2009-07-15 2010-05-26 Vector dual para la inhibición del virus de la inmunodeficiencia humana
ES17197440T Active ES2770627T3 (es) 2009-07-15 2010-05-26 Vector dual para la inhibición del virus de la inmunodeficiencia humana

Family Applications After (2)

Application Number Title Priority Date Filing Date
ES10720980.1T Active ES2546663T3 (es) 2009-07-15 2010-05-26 Vector dual para la inhibición del virus de la inmunodeficiencia humana
ES17197440T Active ES2770627T3 (es) 2009-07-15 2010-05-26 Vector dual para la inhibición del virus de la inmunodeficiencia humana

Country Status (9)

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US (3) US20120201794A1 (es)
EP (4) EP3659435A1 (es)
JP (4) JP5828838B2 (es)
AU (1) AU2010274031B2 (es)
CA (1) CA2767972A1 (es)
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Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8909195D0 (en) 1989-04-22 1989-09-13 Cambridge Consultants Scoring system
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
GB201012410D0 (en) * 2010-07-23 2010-09-08 Medical Res Council Intracellular immunity
CN103841982A (zh) * 2011-06-29 2014-06-04 罗尼路发展有限公司 Hiv感染的预防和治疗
EP3563859B1 (en) 2012-08-13 2021-10-13 Cedars-Sinai Medical Center Cardiosphere-derived exosomes for tissue regeneration
EP3027755B1 (en) * 2013-08-02 2019-10-09 The Regents of The University of California Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors
WO2016014837A1 (en) * 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Gene editing for hiv gene therapy
EP4494699A3 (en) 2014-10-03 2025-04-30 Cedars-Sinai Medical Center Cardiosphere-derived cells and exosomes secreted by such cells in the treatment of muscular dystrophy
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
PL3294893T3 (pl) 2015-05-13 2024-04-29 Csl Behring Gene Therapy, Inc. Bioprodukcja wektorów lentiwirusowych
HK1247963A1 (zh) * 2015-05-18 2018-10-05 Csl贝林基因治疗股份有限公司 用於hiv的治疗的基因疗法及其用途
DK3283658T3 (da) 2015-05-18 2020-05-18 Calimmune Inc Fremgangsmåder til at diskriminere mellem HIV-1 og lentivirus-vektorer
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
CN116064678A (zh) 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
WO2017139065A1 (en) * 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
CN105861559B (zh) * 2016-04-11 2018-05-01 苏州奥特铭医药科技有限公司 一种能直接反应i型干扰素应答的慢病毒载体及其制备方法与应用
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. SYSTEM FOR THE NON-INTEGRAL VIRAL ISSUE AND PROCEDURE RELATING TO IT
CN106086075B (zh) * 2016-06-23 2019-08-06 福建医科大学 CXCR4RNAi慢病毒载体的构建方法
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR THE TREATMENT OF PARKINSON'S DISEASE
JP7260170B2 (ja) * 2017-01-09 2023-04-18 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド 事前の免疫化ステップのないｈｉｖ免疫療法
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JP2020524996A (ja) 2017-06-16 2020-08-27 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド ヒトガンマデルタｔ細胞を介する腫瘍細胞傷害性の活性化のための方法および組成物
CN111164211B (zh) 2017-07-18 2024-08-02 杰特贝林基因治疗股份有限公司 用于治疗β-血红蛋白病的组合物和方法
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
IL274632B2 (en) * 2017-11-15 2024-12-01 Weird Science Llc Methods and compositions for non-myeloablative bone marrow reconstitution
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
CA3106403A1 (en) 2018-07-15 2020-01-23 Enochian BioPharma, Inc. Methods and compositions using recombinant dendritic cells for cancer therapy
US11352646B2 (en) 2018-11-05 2022-06-07 American Gene Technologies International Inc. Vector system for expressing regulatory RNA
AU2022212090A1 (en) * 2021-01-28 2023-07-06 Allogene Therapeutics, Inc. Methods for transducing immune cells
RU2768032C1 (ru) * 2021-09-02 2022-03-23 Федеральное бюджетное учреждение науки "Государственный научный центр вирусологии и биотехнологии "Вектор" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ГНЦ ВБ "Вектор" Роспотребнадзора) Генетическая конструкция для экспрессии генов mNG_CD4-CXCR4, рекомбинантная плазмида rVSV_mNG_CD4-CXCR4 и рекомбинантный штамм вируса везикулярного стоматита rVSV_mNG_CD4-CXCR4, обеспечивающий таргетный виролизис клеток, экспонирующих на своей поверхности белки gp120/gp 41 ВИЧ-1 тропности X4
RU2769125C1 (ru) * 2021-09-02 2022-03-28 Федеральное бюджетное учреждение науки "Государственный научный центр вирусологии и биотехнологии "Вектор" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ГНЦ ВБ "Вектор" Роспотребнадзора) Генетическая конструкция для экспрессии генов mNG_CD4-CCR5, рекомбинантная плазмида rVSV_mNG_CD4-CCR5 и рекомбинантный штамм вируса везикулярного стоматита rVSV_mNG_CD4-CCR5, обеспечивающий таргетный виролизис клеток, экспонирующих на своей поверхности белки gp120/gp41 ВИЧ-1 тропности R5
CA3259099A1 (en) * 2022-06-15 2023-12-21 Cedars-Sinai Medical Center Non-coding RNA agents (BCYRN1 and their derivatives) for treating immune disorders
WO2026039311A1 (en) * 2024-08-10 2026-02-19 Vectorgen Lentiviral vector for treating human immunodeficiency virus infection

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4714680B1 (en) 1984-02-06 1995-06-27 Univ Johns Hopkins Human stem cells
US4965204A (en) 1984-02-06 1990-10-23 The Johns Hopkins University Human stem cells and monoclonal antibodies
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US7737124B2 (en) * 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
US7850990B2 (en) * 2001-10-03 2010-12-14 Celator Pharmaceuticals, Inc. Compositions for delivery of drug combinations
RU2324738C2 (ru) * 2006-06-16 2008-05-20 Федеральное государственное учреждение науки "Государственный научный центр вирусологии и биотехнологии "Вектор" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФГУН ГНЦ ВБ "Вектор" Роспотребнадзора) ГЕНЕТИЧЕСКАЯ КОНСТРУКЦИЯ НА ОСНОВЕ ВЕКТОРНОЙ ПЛАЗМИДЫ pEGFP-N1, ПРОДУЦИРУЮЩАЯ siPHK-ИНГИБИТОРЫ РЕПРОДУКЦИИ ВИРУСА ИММУНОДЕФИЦИТА ЧЕЛОВЕКА 1 ТИПА (ЕЕ ВАРИАНТЫ)
CN101841399B (zh) * 2009-03-16 2013-08-07 上海贝尔股份有限公司 多基站协作接收网络中实现同步上行harq进程的方法和装置
RS53257B2 (sr) * 2009-07-24 2018-06-29 Immune Design Corp Lentiviralni vektori pseudotipizirani sa omotačem od glikobelančevine iz sindbis-virusa

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US20220016176A1 (en) 2022-01-20
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