HUE029977T2 - Oligonucleotides to generate a change in the sequence of target RNA molecules present in a living cell - Google Patents
Oligonucleotides to generate a change in the sequence of target RNA molecules present in a living cell Download PDFInfo
- Publication number
- HUE029977T2 HUE029977T2 HUE13760133A HUE13760133A HUE029977T2 HU E029977 T2 HUE029977 T2 HU E029977T2 HU E13760133 A HUE13760133 A HU E13760133A HU E13760133 A HUE13760133 A HU E13760133A HU E029977 T2 HUE029977 T2 HU E029977T2
- Authority
- HU
- Hungary
- Prior art keywords
- oligonucleotide
- rna
- sequence
- seq
- cftr
- Prior art date
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Classifications
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- 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
- 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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- 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
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
-
- 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
- A61K9/08—Solutions
-
- 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
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- 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
- 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
- C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
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- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Pulmonology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261670681P | 2012-07-12 | 2012-07-12 | |
| US201261718801P | 2012-10-26 | 2012-10-26 | |
| EP13166465 | 2013-05-03 | ||
| EP13172515 | 2013-06-18 | ||
| EP13173818 | 2013-06-26 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE029977T2 true HUE029977T2 (en) | 2017-04-28 |
Family
ID=49916369
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE13760133A HUE029977T2 (en) | 2012-07-12 | 2013-07-12 | Oligonucleotides to generate a change in the sequence of target RNA molecules present in a living cell |
Country Status (23)
| Country | Link |
|---|---|
| US (2) | US9605255B2 (https=) |
| EP (2) | EP2852668B1 (https=) |
| JP (1) | JP6373833B2 (https=) |
| KR (1) | KR20150037968A (https=) |
| CN (1) | CN104640986B (https=) |
| AU (1) | AU2013287353B2 (https=) |
| BR (1) | BR112015000710A8 (https=) |
| CA (1) | CA2878934A1 (https=) |
| CY (1) | CY1117858T1 (https=) |
| DK (2) | DK2852668T3 (https=) |
| ES (2) | ES2698522T3 (https=) |
| HR (1) | HRP20160854T1 (https=) |
| HU (1) | HUE029977T2 (https=) |
| IL (1) | IL236654B (https=) |
| MX (1) | MX356625B (https=) |
| NZ (1) | NZ703824A (https=) |
| PL (1) | PL2852668T3 (https=) |
| PT (1) | PT2852668T (https=) |
| RS (1) | RS54944B1 (https=) |
| RU (1) | RU2663110C2 (https=) |
| SI (1) | SI2852668T1 (https=) |
| SM (1) | SMT201600234B (https=) |
| WO (1) | WO2014011053A1 (https=) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007079139A2 (en) | 2005-12-28 | 2007-07-12 | Vertex Pharmaceuticals, Inc. | Solid forms of n-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide |
| US8563573B2 (en) | 2007-11-02 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Azaindole derivatives as CFTR modulators |
| US8802868B2 (en) | 2010-03-25 | 2014-08-12 | Vertex Pharmaceuticals Incorporated | Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide |
| ES2608474T3 (es) | 2010-04-22 | 2017-04-11 | Vertex Pharmaceuticals Incorporated | Proceso de producción de compuestos indol cycloalkylcarboxamido |
| RS54944B1 (sr) | 2012-07-12 | 2016-11-30 | Proqr Therapeutics Ii Bv | Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji |
| ES2885181T3 (es) | 2014-04-15 | 2021-12-13 | Vertex Pharma | Composiciones farmacéuticas para el tratamiento de enfermedades mediadas por el regulador de la conductancia transmembrana de fibrosis quística |
| KR102589295B1 (ko) | 2014-05-14 | 2023-10-13 | 타르그이뮨 테라퓨틱스 아게 | 개선된 폴리에틸렌이민 폴리에틸렌글리콜 벡터 |
| KR20170063954A (ko) | 2014-10-07 | 2017-06-08 | 버텍스 파마슈티칼스 인코포레이티드 | 낭성 섬유증 막횡단 전도도 조절자의 조정제의 공-결정 |
| GB201418892D0 (en) * | 2014-10-23 | 2014-12-10 | Proqr Therapeutics B V | DNA editing |
| US12359197B2 (en) * | 2014-12-12 | 2025-07-15 | Etagen Pharma, Inc. | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
| EP3234134B1 (en) * | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Targeted rna editing |
| US20210401869A1 (en) * | 2015-02-20 | 2021-12-30 | Rosalind Franklin University Of Medicine And Science | Antisense Compounds Targeting Genes Associated with Cystic Fibrosis |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
| EP3359667A1 (en) | 2015-10-05 | 2018-08-15 | ProQR Therapeutics II B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
| CN108367021A (zh) * | 2015-10-15 | 2018-08-03 | 希望之城 | 包含硫代磷酸化寡脱氧核苷酸的化合物和组合物及其使用方法 |
| CA3022319A1 (en) * | 2016-05-06 | 2017-11-09 | Tod M. Woolf | Improved methods for genome editing with and without programmable nucleases |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| CN110352244B (zh) * | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| CN110612353A (zh) | 2017-03-03 | 2019-12-24 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| CN107267516B (zh) * | 2017-07-28 | 2020-09-29 | 佛山科学技术学院 | 双sgRNA介导的基因精确修饰方法及应用 |
| WO2019076919A1 (en) * | 2017-10-17 | 2019-04-25 | INSERM (Institut National de la Santé et de la Recherche Médicale) | COMBINED TREATMENT OF CYSTIC FIBROSIS |
| WO2019156726A1 (en) * | 2018-02-09 | 2019-08-15 | Ohio State Innovation Foundation | Rna nanostructures, methods of making, and uses thereof |
| JP7478923B2 (ja) | 2018-03-27 | 2024-05-08 | ユニバーシティ オブ ロチェスター | シュードウリジン化のための核酸分子 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| EP3914260A4 (en) | 2019-01-22 | 2023-05-17 | Korro Bio, Inc. | RNA-EDITING OLIGONUCLEOTIDES AND THEIR USE |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| CN113397996A (zh) * | 2021-07-23 | 2021-09-17 | 河南省人民医院 | 一种抗菌漱口水及其制备方法 |
| CN114917227B (zh) * | 2022-05-19 | 2023-10-31 | 中国人民解放军海军军医大学 | 依伐卡托在制备抗蜱传脑炎病毒、西尼罗病毒、黄热病毒和基孔肯雅热病毒感染药中的应用 |
| EP4432289A1 (en) | 2023-03-15 | 2024-09-18 | Shape Therapeutics Inc. | Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HRP930935A2 (en) * | 1992-06-11 | 1994-12-31 | Astra Ab | New dna sequences |
| DE69435005T2 (de) | 1993-05-11 | 2008-04-17 | The University Of North Carolina At Chapel Hill | Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung |
| US6207455B1 (en) | 1997-05-01 | 2001-03-27 | Lung-Ji Chang | Lentiviral vectors |
| IL132463A0 (en) | 1997-05-13 | 2001-03-19 | Univ North Carolina | Lentivirus - based gene transfer vectors |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| US6218181B1 (en) | 1998-03-18 | 2001-04-17 | The Salk Institute For Biological Studies | Retroviral packaging cell line |
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| DE19909769A1 (de) | 1999-03-05 | 2000-09-07 | Bundesrepublik Deutschland Let | Von SIVagm abgeleitete lentivirale Vektoren, Verfahren zu ihrer Herstellung und ihre Verwendung zur Genübertragung in Säugerzellen |
| WO2005094370A2 (en) * | 2004-03-29 | 2005-10-13 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| JP2013518603A (ja) | 2010-02-08 | 2013-05-23 | アイシス ファーマシューティカルズ， インコーポレーテッド | 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 |
| DK2561077T3 (en) * | 2010-04-23 | 2016-08-01 | Arrowhead Res Corp | Organic compositions for the treatment of beta-ENaC-related diseases |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US20150209448A1 (en) | 2012-07-12 | 2015-07-30 | Proqr Therapeutics N.V. | Exon replacement with stabilized artificial rnas |
| RS54944B1 (sr) | 2012-07-12 | 2016-11-30 | Proqr Therapeutics Ii Bv | Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji |
-
2013
- 2013-07-12 RS RS20160489A patent/RS54944B1/sr unknown
- 2013-07-12 EP EP13760133.2A patent/EP2852668B1/en active Active
- 2013-07-12 NZ NZ703824A patent/NZ703824A/en not_active IP Right Cessation
- 2013-07-12 PL PL13760133.2T patent/PL2852668T3/pl unknown
- 2013-07-12 DK DK13760133.2T patent/DK2852668T3/en active
- 2013-07-12 HU HUE13760133A patent/HUE029977T2/en unknown
- 2013-07-12 US US14/414,303 patent/US9605255B2/en not_active Expired - Fee Related
- 2013-07-12 SI SI201330214A patent/SI2852668T1/sl unknown
- 2013-07-12 PT PT137601332T patent/PT2852668T/pt unknown
- 2013-07-12 ES ES16166821T patent/ES2698522T3/es active Active
- 2013-07-12 AU AU2013287353A patent/AU2013287353B2/en not_active Ceased
- 2013-07-12 DK DK16166821.5T patent/DK3103872T3/en active
- 2013-07-12 ES ES13760133.2T patent/ES2584856T3/es active Active
- 2013-07-12 WO PCT/NL2013/050534 patent/WO2014011053A1/en not_active Ceased
- 2013-07-12 EP EP16166821.5A patent/EP3103872B1/en not_active Not-in-force
- 2013-07-12 JP JP2015521573A patent/JP6373833B2/ja not_active Expired - Fee Related
- 2013-07-12 HR HRP20160854TT patent/HRP20160854T1/hr unknown
- 2013-07-12 CN CN201380047636.5A patent/CN104640986B/zh not_active Expired - Fee Related
- 2013-07-12 KR KR1020157002757A patent/KR20150037968A/ko not_active Ceased
- 2013-07-12 MX MX2015000506A patent/MX356625B/es active IP Right Grant
- 2013-07-12 RU RU2015104763A patent/RU2663110C2/ru not_active IP Right Cessation
- 2013-07-12 CA CA2878934A patent/CA2878934A1/en not_active Abandoned
- 2013-07-12 BR BR112015000710A patent/BR112015000710A8/pt active Search and Examination
-
2015
- 2015-01-11 IL IL236654A patent/IL236654B/en not_active IP Right Cessation
-
2016
- 2016-07-15 SM SM201600234T patent/SMT201600234B/it unknown
- 2016-07-22 CY CY20161100722T patent/CY1117858T1/el unknown
-
2017
- 2017-02-10 US US15/430,069 patent/US9994856B2/en not_active Expired - Fee Related
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