IL308824A - Nucleic acid products and methods of administration thereof - Google Patents

Nucleic acid products and methods of administration thereof

Info

Publication number
IL308824A
IL308824A IL308824A IL30882423A IL308824A IL 308824 A IL308824 A IL 308824A IL 308824 A IL308824 A IL 308824A IL 30882423 A IL30882423 A IL 30882423A IL 308824 A IL308824 A IL 308824A
Authority
IL
Israel
Prior art keywords
synthetic rna
gene
rna
seq
disease
Prior art date
Application number
IL308824A
Other languages
English (en)
Hebrew (he)
Inventor
Matthew Angel
Christopher Rohde
Original Assignee
Factor Bioscience Inc
Matthew Angel
Christopher Rohde
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 Factor Bioscience Inc, Matthew Angel, Christopher Rohde filed Critical Factor Bioscience Inc
Publication of IL308824A publication Critical patent/IL308824A/en

Links

Classifications

    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/48—Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003—Peptide-nucleic acids (PNAs)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Emergency Medicine (AREA)
  • Pulmonology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Child & Adolescent Psychology (AREA)
IL308824A 2016-08-17 2017-08-17 Nucleic acid products and methods of administration thereof IL308824A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662376209P 2016-08-17 2016-08-17
US201762509350P 2017-05-22 2017-05-22
PCT/US2017/047440 WO2018035377A1 (en) 2016-08-17 2017-08-17 Nucleic acid products and methods of administration thereof

Publications (1)

Publication Number Publication Date
IL308824A true IL308824A (en) 2024-01-01

Family

ID=61197041

Family Applications (2)

Application Number Title Priority Date Filing Date
IL264439A IL264439B2 (en) 2016-08-17 2017-08-17 A suitable viral preparation containing a synthetic messenger RNA encoding a gene editing protein for use in cancer treatment and a synthetic messenger RNA encoding a gene editing protein for use in therapy
IL308824A IL308824A (en) 2016-08-17 2017-08-17 Nucleic acid products and methods of administration thereof

Family Applications Before (1)

Application Number Title Priority Date Filing Date
IL264439A IL264439B2 (en) 2016-08-17 2017-08-17 A suitable viral preparation containing a synthetic messenger RNA encoding a gene editing protein for use in cancer treatment and a synthetic messenger RNA encoding a gene editing protein for use in therapy

Country Status (8)

Country Link
US (12) US10576167B2 (enExample)
EP (1) EP3500585A4 (enExample)
JP (3) JP2019528284A (enExample)
CN (2) CN109803977B (enExample)
AU (2) AU2017312113B2 (enExample)
CA (1) CA3033788A1 (enExample)
IL (2) IL264439B2 (enExample)
WO (1) WO2018035377A1 (enExample)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8956849B2 (en) * 2007-11-01 2015-02-17 The Board Of Trustees Of The University Of Arkansas Compositions and methods of enhancing immune responses to Eimeria
BR122019025678B1 (pt) * 2012-11-01 2023-04-18 Factor Bioscience Inc Composições que compreendem ácido nucleico que codifica uma proteína de edição gênica
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
US11325948B2 (en) 2016-03-19 2022-05-10 Exuma Biotech Corp. Methods and compositions for genetically modifying lymphocytes to express polypeptides comprising the intracellular domain of MPL
JP2019528284A (ja) * 2016-08-17 2019-10-10 ファクター バイオサイエンス インコーポレイテッド 核酸産物およびその投与方法
EP3596108A4 (en) 2017-03-15 2020-12-23 Pandion Operations, Inc. TARGETED IMMUNOTOLERANCE
CN106987604B (zh) * 2017-03-29 2021-05-28 北京希诺谷生物科技有限公司 一种制备动脉粥样硬化疾病模型犬的方法
JP2020521452A (ja) 2017-05-24 2020-07-27 パンディオン・セラピューティクス・インコーポレイテッド 標的化免疫寛容
KR102636537B1 (ko) 2017-05-31 2024-02-15 울트라제닉스 파마수티컬 인코포레이티드 글리코겐 축적 질환 유형 iii에 대한 치료제
MA50803A (fr) 2017-11-22 2020-09-30 Modernatx Inc Polynucléotides codant pour l'ornithine transcarbamylase pour le traitement de troubles du cycle de l'urée
US10174092B1 (en) 2017-12-06 2019-01-08 Pandion Therapeutics, Inc. IL-2 muteins
US10946068B2 (en) 2017-12-06 2021-03-16 Pandion Operations, Inc. IL-2 muteins and uses thereof
USRE50550E1 (en) 2017-12-06 2025-08-26 Pandion Operations, Inc. IL-2 muteins and uses thereof
US20210024907A1 (en) * 2018-03-27 2021-01-28 Factor Bioscience Inc. Nucleic acid-based therapeutics
US20210371858A1 (en) * 2018-05-11 2021-12-02 Beam Therapeutics Inc. Methods of suppressing pathogenic mutations using programmable base editor systems
CN109468372B (zh) * 2018-11-21 2020-08-04 中国人民解放军陆军军医大学第一附属医院 多种遗传代谢性肝病靶向文库的引物组合、方法及试剂盒
JP7558167B2 (ja) 2018-12-03 2024-09-30 ボード オブ リージェンツ，ザ ユニバーシティ オブ テキサス システム オリゴベンズアミド類似体およびがん処置におけるそれらの使用
WO2020118115A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
CN109750038B (zh) * 2018-12-29 2021-08-31 烟台毓璜顶医院 一种长非编码rna及在制备诊断子痫前期及靶点药物治疗中的应用
CN109628454B (zh) * 2019-01-30 2022-05-24 上海海洋大学 斑马鱼糖原贮积症gys1和gys2基因突变体的构建方法
CN109777832B (zh) * 2019-02-01 2020-08-04 国家卫生健康委科学技术研究所 碱基编辑模拟、修复与甘露糖苷贮积症相关的man2b1c2248t突变的试剂和方法
EP3972992A4 (en) 2019-05-20 2023-07-19 Pandion Operations, Inc. MADCAM TARGETED IMMUNOTLERANCE
JP2022536364A (ja) 2019-06-13 2022-08-15 ザ ジェネラル ホスピタル コーポレイション 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法
CN110250109B (zh) * 2019-07-01 2021-09-24 上海交通大学医学院附属新华医院 乙醛酸代谢异常相关疾病模型的构建方法、组合物及试剂盒和应用
CN114450265B (zh) 2019-07-03 2024-12-24 菲克特生物科学股份有限公司 阳离子脂质及其用途
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
KR20220044212A (ko) 2019-08-09 2022-04-06 넛크래커 테라퓨틱스 인코포레이티드 미세유체 장치 및 이의 사용 방법
JP2022546101A (ja) * 2019-09-01 2022-11-02 エクスマ バイオテック コーポレイション リンパ球の改変および送達のための方法および組成物
CN111100196B (zh) * 2019-11-08 2021-07-13 上海交通大学 一种生物活性多肽qilsvpgwtysr及其制备方法和应用
EP4067495A4 (en) * 2019-11-26 2023-06-14 Korea Research Institute of Bioscience and Biotechnology MRNA CONSTRUCT FOR PROTEIN EXPRESSION AND ITS USE
WO2021168079A1 (en) 2020-02-21 2021-08-26 Pandion Operations, Inc. Tissue targeted immunotolerance with a cd39 effector
CN115515605A (zh) * 2020-02-24 2022-12-23 美国政府(由卫生和人类服务部的部长所代表) 表达造血生长因子受体的nk细胞或t细胞及用于治疗癌症的用途
WO2021173712A1 (en) * 2020-02-24 2021-09-02 The Board Of Regents Of The University Of Texas System Molecular biomarkers and targets for fuchs' endothelial corneal dystrophy and glaucoma
WO2021178448A1 (en) * 2020-03-02 2021-09-10 Motor Life Sciences, Llc Compositions and methods for diagnosing, preventing, and treating amyotrophic lateral sclerosis in patients with hypofunctional anti-trypsin activity
EP4114470A4 (en) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
EP4149252A4 (en) * 2020-05-12 2024-10-02 Factor Bioscience Inc. GENETICALLY ENGINEERED PROTEINS
CN111714619B (zh) * 2020-07-15 2023-03-14 上海市浦东新区人民医院 成纤维生长因子6在制备缓解非酒精性脂肪性肝炎肝损伤的药物中的应用
CA3189438A1 (en) 2020-07-21 2022-01-27 Chembeau LLC Diester cosmetic formulations and uses thereof
KR20230043151A (ko) 2020-07-24 2023-03-30 더 제너럴 하스피탈 코포레이션 증강된 바이러스-유사 입자 및 세포에의 전달을 위한 그의 사용 방법
CN111944094B (zh) * 2020-08-07 2022-01-07 西南石油大学 水凝胶共聚物、油水分离涂料及其制备方法、应用、油水分离网及其制备方法
CN112143789A (zh) * 2020-09-10 2020-12-29 长沙金域医学检验实验室有限公司 一种鉴定galt基因同时含两个突变位点位置的试剂盒及方法
US20240000891A1 (en) * 2020-12-08 2024-01-04 The Board Of Trustees Of The Leland Stanford Junior University Growth and differentiation factor 15 for treatment of proliferative vitreoretinopathy therapy
CN114717229B (zh) * 2021-01-05 2024-09-10 麦塞拿治疗(香港)有限公司 治疗性mRNA的无细胞和无载体体外RNA转录方法和核酸分子
US12364531B2 (en) * 2021-02-16 2025-07-22 RecensMedical, Inc.; Methods for treating skin disorders using precision cooling technology
IL307350A (en) * 2021-04-02 2023-11-01 Krystal Biotech Inc Viral vectors for cancer treatment
CN113151182B (zh) * 2021-04-19 2022-12-06 深圳明杰生物工程有限公司 基于msc的基因重组细胞的制备方法及其应用
US20240207377A1 (en) * 2021-05-03 2024-06-27 Ageronix SA Alpha-1-antitrypsin (aat) in the treatment and/or prevention of neurological disorders
MX2023012647A (es) 2021-05-07 2023-11-08 Helix Nanotechnologies Inc Acidos ribonucleicos modificados y sus usos.
AR128311A1 (es) * 2022-01-21 2024-04-17 Inst Pasteur De Montevideo Inmunización activa para reducir el dolor osteoarticular, neuropático y en cáncer
WO2023225572A2 (en) 2022-05-17 2023-11-23 Nvelop Therapeutics, Inc. Compositions and methods for efficient in vivo delivery
WO2024121160A1 (en) * 2022-12-05 2024-06-13 Ethris Gmbh Regulator(s) of energy homeostasis-encoding rna molecule(s) with increased translation efficiency
WO2024229116A2 (en) * 2023-05-01 2024-11-07 Factor Bioscience Inc. Methods for reprogramming and gene editing cells
CN116585342B (zh) * 2023-05-23 2024-08-16 源生生物科技(青岛)有限责任公司 包含miRNA的活性成分及其应用
CN117248020B (zh) * 2023-09-28 2025-06-27 山东大学 Hmgcl作为胶质瘤诊断/预后标志物和治疗靶点的应用
WO2025155772A1 (en) * 2024-01-19 2025-07-24 Gene Company (Pty)Ltd. Alpha-1 anti-trypsin upregulating polynucleotides and method of use and treatment thereof
CN118716283B (zh) * 2024-07-29 2025-01-28 安徽省农业科学院水产研究所 一种保持遗传多样性的鱼类育种方法

Family Cites Families (184)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US527565A (en) * 1894-10-16 tripp
US3034507A (en) 1960-05-10 1962-05-15 American Cyanamid Co Intracutaneous injection device
US3539465A (en) 1968-10-08 1970-11-10 Ncr Co Encapsulation of hydrophilic liquid-in-oil emulsions
US3675766A (en) 1970-02-04 1972-07-11 Sol Roy Rosenthal Multiple puncture injector device
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
IE61148B1 (en) * 1988-03-10 1994-10-05 Ici Plc Method of detecting nucleotide sequences
US5656593A (en) 1991-03-11 1997-08-12 Creative Biomolecules, Inc. Morphogen induced periodontal tissue regeneration
US6949505B1 (en) 1991-03-11 2005-09-27 Curis, Inc. Morphogen-induced dendritic growth
US6506729B1 (en) 1991-03-11 2003-01-14 Curis, Inc. Methods and compositions for the treatment and prevention of Parkinson's disease
US5674844A (en) 1991-03-11 1997-10-07 Creative Biomolecules, Inc. Treatment to prevent loss of and/or increase bone mass in metabolic bone diseases
US6800603B2 (en) 1991-03-11 2004-10-05 Curis, Inc. Morphogen-induced neural cell adhesion
US5849686A (en) 1991-03-11 1998-12-15 Creative Biomolecules, Inc. Morphogen-induced liver regeneration
US5993434A (en) 1993-04-01 1999-11-30 Genetronics, Inc. Method of treatment using electroporation mediated delivery of drugs and genes
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
US5711964A (en) 1995-06-07 1998-01-27 United States Of America Method for the intracellular delivery of biomolecules using liposomes containing cationic lipids and vitamin D
JP2000507939A (ja) 1996-03-22 2000-06-27 ザ ジェネラル ホスピタル コーポレーション 中枢神経系の虚血または外傷後のポリペプチド成長因子の投与
US6498142B1 (en) 1996-05-06 2002-12-24 Curis, Inc. Morphogen treatment for chronic renal failure
US6602693B1 (en) 1996-07-03 2003-08-05 Clear Solutions Biotech, Inc. Gene encoding hyaluronan synthase
US6413544B1 (en) 1996-08-19 2002-07-02 The United States Of America As Represented By The Department Of Health And Human Services Liposome complexes for increased systemic delivery
US6770291B2 (en) 1996-08-30 2004-08-03 The United States Of America As Represented By The Department Of Health And Human Services Liposome complexes for increased systemic delivery
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
US6653104B2 (en) 1996-10-17 2003-11-25 Immunomedics, Inc. Immunotoxins, comprising an internalizing antibody, directed against malignant and normal cells
WO1998030679A1 (en) 1997-01-10 1998-07-16 Life Technologies, Inc. Embryonic stem cell serum replacement
US6316260B1 (en) 1997-08-22 2001-11-13 Bernina Biosystems Gmbh Tetraether lipid derivatives and liposomes and lipid agglomerates containing tetraether lipid derivatives, and use thereof
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. SENSE mRNA THERAPY
EP1129064B1 (en) 1998-11-12 2008-01-09 Invitrogen Corporation Transfection reagents
GB9902000D0 (en) 1999-01-30 1999-03-17 Delta Biotechnology Ltd Process
WO2000074763A2 (en) 1999-06-04 2000-12-14 Georgia Tech Research Corporation Devices and methods for enhanced microneedle penetration of biological barriers
US7621606B2 (en) 2001-08-27 2009-11-24 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
US20110171185A1 (en) 1999-06-30 2011-07-14 Klimanskaya Irina V Genetically intact induced pluripotent cells or transdifferentiated cells and methods for the production thereof
US20020054895A1 (en) 1999-07-23 2002-05-09 Alwyn Company, Inc. Allantoin-containing skin cream
US6595947B1 (en) 2000-05-22 2003-07-22 Becton, Dickinson And Company Topical delivery of vaccines
DE60132471T2 (de) 2000-09-26 2009-01-15 Idera Pharmaceuticals, Inc., Cambridge Modulation der immunostimulatorischen aktivität von immunostimulierenden oligonukleotidanaloga durch positionelle chemische veränderungen
US7189759B2 (en) 2001-05-23 2007-03-13 Medicis Pharmaceutical Corporation Compositions for the treatment of pigmentation disorders and methods for their manufacture
CA2461185A1 (en) 2001-09-21 2003-04-03 Japan Science And Technology Corporation Method of screening reprogramming factor, reprogramming factor screened by the method, method of using the reprogramming factor, method of differentiating undifferentiated fused cells and method of constructing cell, tissues and organs
EP1444345A4 (en) 2001-10-18 2004-12-08 Ixion Biotechnology Inc TRANSFORMATION OF STEM CELLS AND LIVER PROGENITORS INTO FUNCTIONAL CELLS OF PANCREAS
US7276489B2 (en) 2002-10-24 2007-10-02 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by optimal presentation of 5′ ends
GB0202149D0 (en) 2002-01-30 2002-03-20 Univ Edinburgh Pluripotency determining factors and uses thereof
US20030186249A1 (en) 2002-04-01 2003-10-02 Zairen Sun Human TARPP genes and polypeptides
JP3785508B2 (ja) 2002-04-15 2006-06-14 学校法人慶應義塾 遺伝子治療における免疫応答を解析できる実験モデルマウス
DE10234192B4 (de) 2002-07-26 2009-11-26 Epoplus Gmbh Co.Kg Verwendung von Erythropoetin
US7737182B2 (en) 2002-08-09 2010-06-15 Taisho Pharmaceutical Co., Ltd. Pharmaceuticals for xerosis
WO2005002507A2 (en) 2003-06-03 2005-01-13 Isis Pharmaceuticals, Inc. Modulation of survivin expression
EP1691780A2 (en) 2003-11-21 2006-08-23 Alza Corporation Gene delivery mediated by liposome-dna complex with cleavable peg surface modification
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
WO2006029197A1 (en) 2004-09-08 2006-03-16 Wisconsin Alumni Research Foundation Medium and culture of embryonic stem cells
MX2007002389A (es) 2004-09-08 2009-02-12 Wisconsin Alumni Res Found Cultivo de celulas progenitoras embrionarias humanas.
WO2006047728A2 (en) 2004-10-27 2006-05-04 New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery Bmp gene and fusion protein
CA2595695A1 (en) 2005-01-25 2006-08-03 Cell Therapeutics, Inc. Biologically active protein conjugates comprising (nn[s/t]) peptide repeats and having increased plasma half-life
US7696318B2 (en) 2005-07-13 2010-04-13 Novo Nordisk Health Care Ag Host cell protein knock-out cells for production of therapeutic proteins
CN101273141B (zh) 2005-07-26 2013-03-27 桑格摩生物科学股份有限公司 外源核酸序列的靶向整合和表达
US8323666B2 (en) 2005-08-01 2012-12-04 Allergan, Inc. Botulinum toxin compositions
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP4174179B1 (en) 2005-08-23 2025-05-07 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
WO2007026353A2 (en) 2005-08-29 2007-03-08 Technion Research & Development Foundation Ltd. Media for culturing stem cells
TW200732347A (en) 2005-10-06 2007-09-01 Trophogen Inc VEGF analogs and methods of use
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
NZ569530A (en) 2005-12-13 2011-07-29 Univ Kyoto Nuclear reprogramming factor
EP1966239A1 (de) 2005-12-22 2008-09-10 CSL Behring GmbH Oktanoatreduziertes human albumin
US7658728B2 (en) 2006-01-10 2010-02-09 Yuzhakov Vadim V Microneedle array, patch, and applicator for transdermal drug delivery
DE102006051516A1 (de) 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
GB0623635D0 (en) 2006-11-27 2007-01-03 Stem Cell Sciences Uk Ltd Pluripotent cell growth media
US7785301B2 (en) 2006-11-28 2010-08-31 Vadim V Yuzhakov Tissue conforming microneedle array and patch for transdermal drug delivery or biological fluid collection
AU2007333225B2 (en) 2006-12-08 2014-06-12 Massachusetts Institute Of Technology Delivery of nanoparticles and/or agents to cells
CN101200758A (zh) 2006-12-15 2008-06-18 华中科技大学 一种检测col7a1基因突变的方法及其用途
US10829733B2 (en) 2007-01-04 2020-11-10 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
US20100172882A1 (en) 2007-01-11 2010-07-08 Glazer Peter M Compositions and methods for targeted inactivation of hiv cell surface receptors
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
US8859229B2 (en) 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
KR101516833B1 (ko) 2007-03-23 2015-05-07 위스콘신 얼럼나이 리서어치 화운데이션 체세포 재프로그래밍
AU2008236629B2 (en) 2007-04-07 2014-03-06 Whitehead Institute For Biomedical Research Reprogramming of somatic cells
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
WO2008151845A2 (en) * 2007-06-15 2008-12-18 Charite - Universitätsmedizin Berlin Methods for producing glycosylated human alpha-1 antitrypsin (a1at) in mammalian host cells
KR101532442B1 (ko) 2007-12-10 2015-06-29 고쿠리츠 다이가쿠 호진 교토 다이가쿠 효율적인 핵 초기화 방법
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
US20110021335A1 (en) 2007-12-14 2011-01-27 Pascal Jozef Paul Buskens Sol-gel process with a protected catalyst
CA2708549C (en) 2007-12-21 2014-04-01 Stryker Corporation Bmp mutants with decreased susceptibility to noggin
SG188904A1 (en) 2008-03-17 2013-04-30 Scripps Research Inst Combined chemical and genetic approaches for generation of induced pluripotent stem cells
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
JP2011160661A (ja) 2008-06-02 2011-08-25 Kyowa Hakko Kirin Co Ltd 血球細胞の初期化法
EP2297307B1 (en) 2008-06-04 2016-06-01 Cellular Dynamics International, Inc. Methods for the production of ips cells using non-viral approach
WO2009147400A1 (en) 2008-06-05 2009-12-10 Iti Scotland Limited Stem cell culture media and methods
WO2010008486A2 (en) 2008-06-24 2010-01-21 Parkinsons Institute Pluripotent cell lines and methods of use thereof
US20100184033A1 (en) 2008-07-16 2010-07-22 West Michael D Methods to accelerate the isolation of novel cell strains from pluripotent stem cells and cells obtained thereby
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
EP2326711A2 (en) 2008-08-20 2011-06-01 VIRxSYS Corporation Compositions and methods for generation of pluripotent stem cells
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
EP3450545B1 (en) 2008-10-24 2023-08-23 Wisconsin Alumni Research Foundation Pluripotent stem cells obtained by non-viral reprogramming
JP2012509106A (ja) 2008-11-18 2012-04-19 スリーエム イノベイティブ プロパティズ カンパニー 中空のマイクロニードルアレイ及び方法
ES2561949T3 (es) 2008-11-21 2016-03-01 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramación de células hacia un estado pluripotente
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP2412800A1 (en) 2010-07-29 2012-02-01 Koninklijke Nederlandse Akademie van Wetenschappen Liver organoid, uses thereof and culture method for obtaining them
WO2010093655A2 (en) 2009-02-10 2010-08-19 University Of Dayton Enhanced method for producing stem-like cells from somatic cells
US10894944B2 (en) 2009-04-10 2021-01-19 Monash University Cell culture media
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
ES2849181T3 (es) 2009-04-22 2021-08-16 Viacyte Inc Composiciones celulares derivadas de células reprogramadas desdiferenciadas
EP2251437B1 (en) 2009-05-13 2013-12-04 Hannelore Breitenbach-Koller Method for identifying compounds that control the translational activity of ribosomal proteins in differential mRNA expression
US8496941B2 (en) 2009-06-03 2013-07-30 National Institute Of Advanced Industrial Science And Technology Vectors for generating pluripotent stem cells and methods of producing pluripotent stem cells using the same
US20120171170A1 (en) 2009-06-16 2012-07-05 Opko Curna, Llc Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
EA022786B1 (ru) 2009-07-31 2016-03-31 Этрис Гмбх Рнк с комбинацией из немодифицированных и модифицированных нуклеотидов для экспрессии белков
US9295697B2 (en) 2009-11-04 2016-03-29 Cellular Dynamics International, Inc. Episomal reprogramming with chemicals
EP2499239A1 (en) 2009-11-11 2012-09-19 Sanford-Burnham Medical Research Institute METHOD FOR GENERATION AND REGULATION OF iPS CELLS AND COMPOSITIONS THEREOF
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
CN114317612B (zh) 2009-12-07 2024-10-29 宾夕法尼亚州大学信托人 用于重编程细胞的包含纯化的经修饰的rna的rna制剂
DK2510096T4 (en) 2009-12-10 2018-05-14 Univ Iowa State Res Found Inc NUMBER EFFECTOR-MEDIATED DNA MODIFICATION
US8557972B2 (en) 2009-12-21 2013-10-15 University Of Washington Through Its Center For Commercialization Compositions and methods for transfection of RNA and controlled stabilization of transfected RNA
CA2788635A1 (en) 2010-02-01 2011-08-04 The Board Of Trustees Of The Leland Stanford Junior University Enhanced efficiency of induced pluripotent stem cell generation
US20140194482A1 (en) 2010-02-02 2014-07-10 Scioderm, Inc. Compositions and methods of treatment of inflammatory skin conditions using allantoin
WO2011102532A1 (ja) 2010-02-16 2011-08-25 国立大学法人九州大学 誘導肝細胞
WO2011110886A1 (en) 2010-03-09 2011-09-15 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
CN102234627B (zh) 2010-04-30 2015-06-03 中国科学院广州生物医药与健康研究院 一种培养基添加剂及其应用
WO2011140397A2 (en) 2010-05-05 2011-11-10 The Regents Of The University Of California Office Of The President Stem cell defined media for xeno-free and feeder free conditions and uses thereof
CA2799095A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
KR101953237B1 (ko) 2010-05-17 2019-02-28 상가모 테라퓨틱스, 인코포레이티드 신규 dna 결합 단백질 및 이의 용도
EP2392208B1 (en) 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
AU2011265733B2 (en) 2010-06-14 2014-04-17 Iowa State University Research Foundation, Inc. Nuclease activity of TAL effector and Foki fusion protein
WO2012019122A2 (en) 2010-08-05 2012-02-09 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
WO2012021632A2 (en) 2010-08-10 2012-02-16 The Johns Hopkins University Generation and use of pluripotent stem cells
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
JP5794588B2 (ja) 2010-09-14 2015-10-14 国立大学法人京都大学 効率的な人工多能性幹細胞の樹立方法
HUE069586T2 (hu) 2010-10-01 2025-03-28 Modernatx Inc Módosított nukleozidok, nukleotidok és nukleinsavak, valamint ezek felhasználása
EP2625278A1 (en) 2010-10-08 2013-08-14 Regents of the University of Minnesota A method to increase gene targeting frequency
WO2012060473A1 (en) 2010-11-04 2012-05-10 Kyoto University Method of efficiently establishing induced pluripotent stem cells
US9267123B2 (en) 2011-01-05 2016-02-23 Sangamo Biosciences, Inc. Methods and compositions for gene correction
CA2832807A1 (en) 2011-03-07 2012-09-13 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
WO2012131090A1 (en) 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
WO2012138453A1 (en) 2011-04-03 2012-10-11 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
WO2012138927A2 (en) 2011-04-05 2012-10-11 Philippe Duchateau Method for the generation of compact tale-nucleases and uses thereof
SG194089A1 (en) * 2011-04-27 2013-11-29 Amyris Inc Methods for genomic modification
WO2012174224A2 (en) 2011-06-17 2012-12-20 Calando Pharmaceuticals, Inc. Methods for administering nucleic acid-based therapeutics
WO2012176015A1 (en) 2011-06-24 2012-12-27 Leo Pharma A/S Methods for treating uv-damaged skin and scc tumors and for removing tattoos with topical ingenol mebutate
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
JP2014520551A (ja) 2011-07-11 2014-08-25 セルラー ダイナミクス インターナショナル， インコーポレイテッド 細胞のリプログラミング方法およびゲノムの改変方法
AU2012289916B2 (en) 2011-08-03 2017-08-03 Phoenix Tissue Repair, Inc. Collagen 7 and related methods
CA3186126A1 (en) * 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
DK3682905T3 (da) 2011-10-03 2022-02-28 Modernatx Inc Modificerede nukleosider, nukleotider og nukleinsyrer og anvendelser deraf
ES2670495T3 (es) 2011-10-11 2018-05-30 Inserm - Institut National De La Santé Et De La Recherche Médicale Tratamiento de salto de exones para epidermólisis ampollosa distrófica
CA2872033A1 (en) 2011-10-11 2013-04-18 Novartis Ag Recombinant self-replicating polycistronic rna molecules
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
MX382822B (es) 2011-12-05 2025-03-13 Factor Bioscience Inc Metodos y productos para transfeccion de celulas.
US20140343129A1 (en) 2011-12-14 2014-11-20 Moderna Therapeutics, Inc. Modified nucleic acids, and acute care uses thereof
HRP20220717T1 (hr) 2011-12-16 2022-07-22 Modernatx, Inc. Modificirani pripravci mrna
RU2014129863A (ru) 2011-12-21 2016-02-10 Модерна Терапьютикс, Инк. Способы повышения жизнеспособности или увеличения продолжительности жизни органа или экспланта органа
EP3421601B1 (en) 2011-12-30 2019-12-04 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
WO2013142514A1 (en) * 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
JP6189415B2 (ja) 2012-04-02 2017-08-30 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
EP2838541A1 (en) 2012-04-20 2015-02-25 Agency For Science, Technology And Research Rnai-based therapies for cardiomyopathies, muscular dystrophies and laminopathies
WO2013163296A1 (en) 2012-04-24 2013-10-31 The Brigham And Women's Hospital, Inc. Generating pluripotent cells de novo
JP6448531B2 (ja) 2012-05-13 2019-01-09 アリール バイオテクノロジー アンド ファーマシューティカルズ， インコーポレイテッド 合成メッセンジャーｒｎａを使用したヒト人工多能性幹細胞のフィーダーフリー誘導
US10119150B2 (en) 2012-05-13 2018-11-06 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free Derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US10155929B2 (en) 2012-05-13 2018-12-18 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US20140031295A1 (en) 2012-07-19 2014-01-30 Lotus Tissue Repair, Inc. Recombinant C7 and Methods of Use
JP6401704B2 (ja) * 2012-10-10 2018-10-10 サンガモ セラピューティクス， インコーポレイテッド Ｔ細胞を修飾する化合物およびその使用
BR122019025678B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Composições que compreendem ácido nucleico que codifica uma proteína de edição gênica
LT2959005T (lt) 2013-02-22 2022-01-25 The Board Of Trustees Of The Leland Stanford Junior University Medicininis naudojimas, susijęs su telomerų ilgėjimu
AU2014220957A1 (en) * 2013-02-22 2015-07-30 Curevac Ag Combination of vaccination and inhibition of the PD-1 pathway
CN105229144A (zh) 2013-02-22 2016-01-06 细胞动力学国际有限公司 通过组合的遗传工程和化学工程经由正向编程产生肝细胞
WO2014134412A1 (en) 2013-03-01 2014-09-04 Regents Of The University Of Minnesota Talen-based gene correction
BR112015031608A2 (pt) * 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
EP4012031A1 (en) 2013-07-03 2022-06-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
US20160220613A1 (en) 2013-09-16 2016-08-04 Agency For Science, Technology And Research Method
US9840699B2 (en) * 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
CN106456793B (zh) 2013-12-12 2021-04-30 生命技术公司 增强转染的膜穿透性肽和组合物以及其使用方法
US20150167017A1 (en) 2013-12-13 2015-06-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
ES2787198T3 (es) * 2014-01-31 2020-10-15 Factor Bioscience Inc ARN sintético para su uso en el tratamiento de la epidermólisis ampollosa distrófica
EP3505623B1 (en) * 2014-02-14 2025-12-24 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
KR102827558B1 (ko) * 2014-04-18 2025-07-11 에디타스 메디신, 인코포레이티드 암 면역요법을 위한 crispr-cas-관련 방법, 조성물 및 구성성분
HUE069802T2 (hu) * 2014-04-23 2025-04-28 Modernatx Inc Nukleinsav vakcinák
CA3197849A1 (en) * 2014-12-29 2016-07-07 Novartis Ag Methods of making chimeric antigen receptor-expressing cells
CA2976376A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
US11851653B2 (en) * 2015-12-01 2023-12-26 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
EP3423580A1 (en) * 2016-03-04 2019-01-09 Editas Medicine, Inc. Crispr-cpf1-related methods, compositions and components for cancer immunotherapy
WO2017165862A1 (en) * 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
JP2019528284A (ja) * 2016-08-17 2019-10-10 ファクター バイオサイエンス インコーポレイテッド 核酸産物およびその投与方法

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