ES2564185T3 - Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos - Google Patents
Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismosInfo
- Publication number
- ES2564185T3 ES2564185T3 ES10004274.6T ES10004274T ES2564185T3 ES 2564185 T3 ES2564185 T3 ES 2564185T3 ES 10004274 T ES10004274 T ES 10004274T ES 2564185 T3 ES2564185 T3 ES 2564185T3
- Authority
- ES
- Spain
- Prior art keywords
- omission
- coding
- oligonucleotide
- exon
- exonic
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
Links
Classifications
-
- 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
-
- 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
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/33—Chemical structure of the base
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Abstract
Un oligonucleótido no codificante aislado que se une al ARN premensajero de la distrofina humana en el que dicho oligonucleótido tiene de 20 a 31 nucleótidos de longitud y es un oligonucleótido que es hibridable de forma específica con una región diana del exón 53 del gen de distrofina, designada como sitio de apareamiento H53A (+23+47), sitio de apareamiento H53A (+39+69), o ambos, en el que dicho oligonucleótido no codificante es un oligonucleótido morfolino no codificante, y, en el que dicho oligonucleótido induce la omisión del exón 53.
Description
5
10
15
20
25
30
35
40
45
50
55
60
65
La frase "farmacéuticamente aceptable" se refiere a entidades y composiciones moleculares que son fisiológicamente tolerables y no producen típicamente una reacción alérgica o u otra de tipo adverso semejante, tal como molestia gástrica y similar, cuando se administra a un paciente. El término "vehículo" se refiere a un diluyente, adyuvante, excipiente., o vehículo con el cual se administra el compuesto. Dichos vehículos farmacéuticos pueden ser líquidos estériles, tales como agua y aceites, incluyendo los de origen del petróleo, animal, vegetal o sintético, tal como aceite de cacahuete, aceite de soja, aceite mineral, aceite de sésamo y similares. Las soluciones de agua o salinas y las soluciones acuosas de dextrosa y glicerol se emplean preferentemente como vehículos, concretamente para solución inyectable. En Martin, Remington's Pharmaceutical Sciences, 18ª Ed., Mack Publishing Co., Easton, PA, (1990) se describen vehículos farmacéuticos adecuados.
En una forma más específica de la invención hay composiciones farmacéuticas proporcionadas que comprenden cantidades terapéuticamente eficaces de una molécula no codificante junto con diluyentes, conservantes, solubilizadores, emulsionantes, adyuvantes y/o vehículos farmacéuticamente aceptables. Dichas composiciones incluyen diluyentes con diverso contenido de tampones (p. ej., Tris-HCl, acetato, fosfato), pH y fuerza iónica, y aditivos tales como detergentes y agentes solubilizadores (p. ej., Tween 80, Polisorbato 80), antioxidantes (p. ej., ácido ascórbico, metabisulfito sódico, conservantes (p. ej., tiomerosal, alcohol bencílico) y sustancias espesantes (p. ej., lactosa, manitol). El material puede incorporarse dentro de preparaciones particuladas de compuestos poliméricos tales como ácido poliláctico, ácido poliglicólico, etc. o dentro de liposomas. También puede usarse ácido hialurónico. Dichas composiciones pueden influir sobre el estado físico, la estabilidad, el índice de liberación in vivo, y el índice de eliminación in vivo de las proteínas y derivados presentes. Véase, p. ej., Martin, Remington's Pharmaceutical Sciences, 18ª Ed. (1990, Mack Publishing Co., Easton, PA 18042) páginas1435-1712. Las composiciones líquidas pueden prepararse en forma líquida, o pueden estar en polvo deshidratado, tal como en forma liofilizada.
Se apreciará que las composiciones farmacéuticas proporcionadas de acuerdo con la presente invención puedan administrarse mediante cualquiera de los medios conocidos en la técnica. Preferentemente, las composiciones farmacéuticas para su administración se administran mediante inyección, por vía oral, o mediante vía pulmonar o nasal. Las moléculas no codificantes se suministran más preferentemente mediante las vías de administración intravenosa, intrarterial, intraperitoneal, intramuscular, o subcutánea.
Terapia basada en molécula no codificante
La presente invención también aborda el uso de las moléculas no codificantes de la presente invención, para la fabricación de un medicamento para la modulación de una enfermedad genética.
El suministro de una cantidad terapéuticamente útil de moléculas no codificantes puede lograrse mediante métodos publicados previamente. Por ejemplo, el suministro intracelular de la molécula no codificante puede ser a través de una composición que comprende una mezcla de la molécula no codificante y una cantidad eficaz de un copolímero en bloque. Un ejemplo de este método se describe en la solicitud de patente de EE.UU. US 20040248833.
Otros métodos de suministro de moléculas no codificantes en el núcleo se describen en Mann CJ y col., (2001) ["Antisense-induced exon skipping and the synthesis of dystrophin in the mdx mouse". Proc., Natl. Acad. Science, 98(1) 42-47] y en Gebski y col., (2003). Human Molecular Genetics, 12(15): 1801-1811.
En la patente de EE.UU. US 6.806.084 se describe un método para introducir una molécula de ácido nucleico dentro de una célula por medio de un vector de expresión, bien como ADN desnudo, o bien en complejo con vehículos lipídicos.
Puede ser deseable suministrar la molécula no codificante en un sistema de dispersión coloidal. Los sistemas de dispersión coloidal incluyen complejos de macromoléculas, nanocápsulas, microesferas, perlas, y sistemas a base de lípidos incluyendo emulsiones de aceite en agua, micelas, micelas mixtas, y liposomas o formulaciones de liposomas.
Los liposomas son vesículas de membrana artificial que son útiles como vehículos de suministro in vitro e in vivo. Estas formulaciones pueden tener características de carga neta catiónica, aniónica o neutra y son características útiles con los métodos de suministro in vitro, in vivo y ex vivo. Se ha demostrado que las vesículas unilaminares grandes (VUG), cuyo tamaño oscila desde 0,2-4,0 PHI.m pueden encapsular un porcentaje sustancial de un tampón acuoso que contiene macromoléculas grandes. El ARN y el ADN pueden encapsularse dentro del interior acuoso y suministrarse a las células en una forma biológicamente activa (Fraley, y col., Trends Biochem. Sci., 6:77, 1981).
Para que un liposoma sea un vehículo de transferencia génica eficaz, deberían estar presentes las características siguientes: (1) encapsulación de la molécula no codificante de interés con una alta eficacia, al tiempo que no se compromete su actividad biológica. (2) unión a una célula diana preferente y sustancial en comparación con células no diana; (3) suministro del contenido acuoso de la vesícula al citoplasma de la célula diana con una alta eficacia; y
(4) expresión precisa y eficaz de la información genética (Mannino, y col., Biotechniques, 6:682, 1988).
10
más cortos o de exón omitido tienden a amplificarse de forma más eficaz y pueden sesgar la estimación del transcrito normal e inducido.
Los tamaños de los productos de la reacción de amplificación se estimaron en un gel de agarosa y se compararon 5 frente a patrones de tamaño apropiados. La confirmación final de la identidad de estos productos se llevó a cabo mediante secuenciación directa del ADN para establecer que se habían mantenido las conexiones de exón correctas
o esperadas.
Una vez se ha inducido la omisión exónica eficaz con una molécula no codificante, las moléculas no codificantes
10 solapantes posteriores pueden sintetizarse y evaluarse después en el ensayo como se ha descrito anteriormente. Nuestra definición de una molécula no codificante eficaz es una que induce omisión exónica fuerte y mantenida a concentraciones de transfección en el orden de 300 nM o menos.
Oligonucleótidos no codificantes dirigidos al exón 53
15 Se prepararon oligonucleótidos no codificantes dirigidos hacia el exón 53 y se ensayó su capacidad de inducir omisión exónica en células musculares humanas usando métodos similares a los descritos anteriormente.
La figura 3 también muestra la molécula no codificante H53A(+39+69) [SEC ID Nº:193] dirigida al sitio aceptor de
20 corte y empalme del exón 53. Este oligonucleótido no codificante fue capaz de inducir omisión del exón 53 a 5, 100, 300 y 600 nM. También se ensayó un "cóctel" de tres oligonucleótidos no codificantes del exón 53:-H53D(+23+47) [SEC ID Nº:195], H53A(+150+175) [SEC ID Nº:196] y H53A(+14-07) [SEC ID Nº:194], como se muestra en la figura 3, y mostraron capacidad para inducir omisión exónica.
25 La tabla 2 a continuación incluye otras moléculas no codificantes ensayadas en un intervalo de concentración de 50, 100, 300 y 600 nM. Estas moléculas no codificantes mostraron una capacidad variable para inducir la omisión del exón 23. La molécula no codificante H53A(+39+69) [SEC ID Nº:193] indujo la omisión más fuerte del exón 53.
Tabla 2 30
- Nombre del oligonucleótido no codificante
- Secuencia Capacidad de inducir omisión
- H53A(+45+69)
- CAU UCA ACU GUU GCC UCC GGU UCU G Omisión débil a 50 nM
- H53A(+39+62)
- CUG UUG CCU CCG GUU CUG AAG GUG Omisión débil a 50 nM
- H53A(+39+69)
-
imagen11 Omisión fuerte a 50 nM
- H53D(+14-07)
- UAC UAA CCU UGG UUU CUG UGA Omisión muy débil a 50 nM
- H53A(+23+47)
- CUG AAG GUG UUC UUG UAC UUC AUC C Omisión muy débil a 50 nM
- H53A(+150+176)
-
imagen12 Omisión muy débil a 50 nM
- H53D(+20-05)
- CUA ACC UUG GUU UCU GUG AUU UUC U Aún sin hacer
- H53D(+09-18)
-
imagen13 Débil a 600 nM
- H53A(-12+10)
- AUU CUU UCA ACU AGA AUA AAA G Sin omisión
- H53A(-07+18)
- GAU UCU GAA UUC UUU CAA CUA GAA U Sin omisión
- H53A(+07+26)
- AUC CCA CUG AUU CUG AAU UC Sin omisión
- H53A(+124+145)
- UUG GCU CUG GCC UGU CCU AAG A Sin omisión
La invención se define con referencia a las siguientes cláusulas: Cláusula 1: Una molécula no codificante capaz de unirse a un sitio diana seleccionado para inducir omisión exónica 35 en el gen de la distrofina, como se ha expuesto en las SEC ID Nº 1 a 202. Cláusula 2: Una molécula no codificante de acuerdo con la cláusula 1, capaz de inducir omisión exónica en los exones 3, 4, 8, 10 a 16, 19 a 40, 42 a 44, 46, 47 y 50 a 53 del gen de la distrofina. 13
Claims (1)
-
imagen1
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
AU2004903474 | 2004-06-28 | ||
AU2004903474A AU2004903474A0 (en) | 2004-06-28 | Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
ES2564185T3 true ES2564185T3 (es) | 2016-03-18 |
Family
ID=35781530
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ES05754344T Active ES2361325T3 (es) | 2004-06-28 | 2005-06-28 | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
ES10004274.6T Active ES2564185T3 (es) | 2004-06-28 | 2005-06-28 | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
ES12162995.0T Active ES2627917T3 (es) | 2004-06-28 | 2005-06-28 | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ES05754344T Active ES2361325T3 (es) | 2004-06-28 | 2005-06-28 | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ES12162995.0T Active ES2627917T3 (es) | 2004-06-28 | 2005-06-28 | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
Country Status (14)
Country | Link |
---|---|
US (47) | US7807816B2 (es) |
EP (9) | EP2206781B1 (es) |
AT (1) | ATE498685T1 (es) |
CY (2) | CY1111447T1 (es) |
DE (1) | DE602005026386D1 (es) |
DK (2) | DK1766010T3 (es) |
ES (3) | ES2361325T3 (es) |
HK (2) | HK1216545A1 (es) |
HR (2) | HRP20110352T1 (es) |
HU (1) | HUE028632T2 (es) |
PL (2) | PL2206781T3 (es) |
PT (1) | PT1766010E (es) |
SI (2) | SI2206781T1 (es) |
WO (1) | WO2006000057A1 (es) |
Families Citing this family (170)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU5752601A (en) | 2000-05-04 | 2001-11-12 | Avi Biopharma Inc | Splice-region antisense composition and method |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
WO2004083432A1 (en) | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
DE602004028930D1 (de) | 2003-04-29 | 2010-10-14 | Avi Biopharma Inc | Zusammensetzungen zur verbesserung der antisense-wirksamkeit und des transports von nukleinsäureanalog in zellen |
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 |
ES2361325T3 (es) | 2004-06-28 | 2011-06-16 | The University Of Western Australia | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, 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 |
WO2007123391A1 (en) | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
PL2049664T3 (pl) | 2006-08-11 | 2012-02-29 | Biomarin Tech Bv | Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA |
CA2981308C (en) * | 2006-09-21 | 2020-12-22 | University Of Rochester | Compositions and methods related to protein displacement therapy for myotonic dystrophy |
WO2009005793A2 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
DK2167136T3 (en) | 2007-07-12 | 2016-07-25 | Biomarin Tech Bv | Molecules for targeting compounds at different selected organs or tissues |
CN101790385A (zh) | 2007-07-12 | 2010-07-28 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子 |
AU2008317566B2 (en) * | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
WO2009086469A2 (en) | 2007-12-28 | 2009-07-09 | Avi Biopharma, Inc. | Immunomodulatory agents and methods of use |
NZ587178A (en) | 2008-02-08 | 2011-11-25 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
WO2009101399A1 (en) * | 2008-02-12 | 2009-08-20 | Isis Innovation Limited | Treatment of muscular dystrophy using peptide nucleic acid ( pna) |
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 |
US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
PL3133160T3 (pl) * | 2008-10-24 | 2019-06-28 | Sarepta Therapeutics, Inc. | Kompozycje do pomijania eksonów w DMD |
ES2562658T3 (es) * | 2008-10-27 | 2016-03-07 | Biomarin Technologies B.V. | Procedimientos y medios para el salto eficiente del exón 45 en el pre-ARNm de la distrofia muscular de Duchenne |
ES2616051T3 (es) | 2008-12-02 | 2017-06-09 | Wave Life Sciences Japan, Inc. | Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo |
WO2010115993A1 (en) * | 2009-04-10 | 2010-10-14 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
US20120046342A1 (en) * | 2009-04-24 | 2012-02-23 | Prosensa 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 |
ITTO20090487A1 (it) * | 2009-06-26 | 2010-12-27 | Univ Ferrara | Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) |
JP5998326B2 (ja) | 2009-07-06 | 2016-09-28 | ウェイブ ライフ サイエンス リミテッドWave Life Sciences Ltd. | 新規核酸プロドラッグおよびその使用方法 |
EP2473607A2 (en) * | 2009-08-31 | 2012-07-11 | INSERM - Institut National de la Santé et de la Recherche Médicale | Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy |
US20120270930A1 (en) | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
KR102239374B1 (ko) * | 2009-11-12 | 2021-04-14 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
MX369004B (es) | 2009-12-18 | 2019-10-24 | Novartis Ag | Composiciones orgánicas para tratar las enfermedades relacionadas con hsf1. |
AU2014280918B2 (en) * | 2009-12-18 | 2016-11-17 | Arrowhead Pharmaceuticals, Inc. | Organic compositions to treat HSF1-related diseases |
WO2011078797A2 (en) * | 2009-12-22 | 2011-06-30 | Singapore Health Services Pte. Ltd | Antisense oligonucleotides and uses threreof |
CA2785451C (en) | 2009-12-24 | 2019-01-22 | Prosensa Technologies B.V. | Molecule for treating an inflammatory disorder |
US9050373B2 (en) | 2010-05-13 | 2015-06-09 | The Charlotte-Mecklenburg Hospital Authority | Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same |
JP2013530154A (ja) | 2010-05-28 | 2013-07-25 | サレプタ セラピューティクス, インコーポレイテッド | 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ |
IT1400425B1 (it) * | 2010-06-08 | 2013-05-31 | Amsterdam Molecular Therapeutics Bv | Modified snrnas for use in therapy. |
EP2601294B1 (en) * | 2010-08-05 | 2018-11-28 | Academisch Ziekenhuis Leiden h.o.d.n. LUMC | Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
EP2426203B1 (en) | 2010-09-02 | 2016-07-13 | Université de Mons | Agents useful in treating facioscapulohumeral muscular dystrophy |
US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, Inc. | dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups |
US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
EP3260540A1 (en) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
EP2672977A1 (en) | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense oligonucleotides |
US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
KR102339196B1 (ko) | 2011-05-05 | 2021-12-15 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
WO2013033407A2 (en) | 2011-08-30 | 2013-03-07 | The Regents Of The University Of California | Identification of small molecules that enhance therapeutic exon skipping |
US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
EP3279324B1 (en) | 2011-10-11 | 2021-02-17 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Exon skipping therapy for dystrophic epidermolysis bullosa |
GB201117880D0 (en) * | 2011-10-17 | 2011-11-30 | Ucl Business Plc | Antisense oligonucleotides |
JP2015500204A (ja) | 2011-11-18 | 2015-01-05 | サレプタ セラピューティクス, インコーポレイテッド | 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット |
IN2014DN06220A (es) * | 2011-12-28 | 2015-10-23 | Nippon Shinyaku Co Ltd | |
JP2015509922A (ja) * | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
NZ700399A (en) | 2012-03-20 | 2016-07-29 | Sarepta Therapeutics Inc | Boronic acid conjugates of oligonucleotide analogues |
DE102012103041A1 (de) | 2012-04-10 | 2013-10-10 | Eberhard-Karls-Universität Tübingen Universitätsklinikum | Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden |
US9372901B2 (en) * | 2012-04-27 | 2016-06-21 | Quixey, Inc. | Searching for software applications based on application attributes |
EP3597741A1 (en) | 2012-04-27 | 2020-01-22 | Duke University | Genetic correction of mutated genes |
EP2850186B1 (en) | 2012-05-16 | 2018-12-19 | Translate Bio MA, Inc. | Compositions and methods for modulating smn gene family expression |
US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
EP3608407A1 (en) * | 2012-07-03 | 2020-02-12 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
PL2872147T3 (pl) | 2012-07-13 | 2023-09-25 | Wave Life Sciences Ltd. | Sposób wytwarzania chiralnych oligonukleotydów |
SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
SI2880053T1 (sl) | 2012-08-01 | 2020-08-31 | Ikaika Therapeutics, Llc | Blažitev poškodbe tkiva in fibroze s protitelesom proti-LTBP4 |
KR20150099804A (ko) | 2012-12-20 | 2015-09-01 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물 |
JP6449231B2 (ja) | 2013-03-14 | 2019-01-09 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
PL2970964T3 (pl) | 2013-03-14 | 2019-06-28 | Sarepta Therapeutics, Inc. | Kompozycje pomijające eksony do leczenia dystrofii mięśniowej |
KR20200139271A (ko) | 2013-03-15 | 2020-12-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 개선된 조성물 |
KR20230136697A (ko) * | 2013-06-05 | 2023-09-26 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
WO2015051283A1 (en) | 2013-10-04 | 2015-04-09 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
SG10201912897UA (en) | 2014-01-16 | 2020-02-27 | Wave Life Sciences Ltd | Chiral design |
MX2016011538A (es) * | 2014-03-12 | 2017-04-13 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido. |
WO2015171918A2 (en) * | 2014-05-07 | 2015-11-12 | Louisiana State University And Agricultural And Mechanical College | Compositions and uses for treatment thereof |
CA2950876A1 (en) | 2014-06-10 | 2015-12-17 | Erasmus University Medical Center Rotterdam | Methods for characterizing alternatively or aberrantly spliced mrna isoforms |
PT3159409T (pt) * | 2014-06-17 | 2020-01-21 | Nippon Shinyaku Co Ltd | Ácido nucleico anti-sentido para utilização no tratamento da distrofia muscular de duchenne |
KR20240123409A (ko) | 2014-09-07 | 2024-08-13 | 셀렉타 바이오사이언시즈, 인크. | 유전자 편집 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물 |
WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
US10525076B2 (en) | 2015-02-20 | 2020-01-07 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting genes associated with cystic fibrosis |
GB201503408D0 (en) | 2015-02-27 | 2015-04-15 | Proqr Therapeutics N V | Oligonucleotides |
GB201504124D0 (en) | 2015-03-11 | 2015-04-22 | Proqr Therapeutics B V | Oligonucleotides |
US10900036B2 (en) | 2015-03-17 | 2021-01-26 | The General Hospital Corporation | RNA interactome of polycomb repressive complex 1 (PRC1) |
MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
PL3283500T3 (pl) | 2015-04-08 | 2021-05-31 | The University Of Chicago | Kompozycje i sposoby korygowania dystrofii mięśniowej obręczowo-kończynowej typu 2c z użyciem pominięcia egzonu |
US10324930B2 (en) * | 2015-05-27 | 2019-06-18 | Sap Se | Database calculation engine with nested multiprovider merging |
US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
WO2017035416A2 (en) | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
PT3351633T (pt) | 2015-09-15 | 2020-07-29 | Nippon Shinyaku Co Ltd | Ácido nucleico antissentido |
WO2017047741A1 (ja) * | 2015-09-16 | 2017-03-23 | 日本新薬株式会社 | 筋萎縮症治療用アンチセンス核酸 |
JP6282620B2 (ja) * | 2015-09-28 | 2018-02-21 | 東芝テック株式会社 | 商品販売データ処理装置及びプログラム |
EP3359668A4 (en) | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
CN108137492B (zh) | 2015-10-09 | 2021-12-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
WO2017136435A1 (en) | 2016-02-01 | 2017-08-10 | The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health | Compounds for modulating fc-epsilon-ri-beta expression and uses thereof |
MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
SG11201809494VA (en) | 2016-05-24 | 2018-12-28 | Sarepta Therapeutics Inc | Pharmaceutical composition comprising eteplirsen |
CN109563114B (zh) | 2016-05-24 | 2022-08-12 | 萨勒普塔医疗公司 | 用于制备寡聚物的方法 |
US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
EP3464306B1 (en) | 2016-05-24 | 2024-03-27 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
CN109311919A (zh) | 2016-06-30 | 2019-02-05 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体 |
WO2018009547A1 (en) | 2016-07-05 | 2018-01-11 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Diagnosing col6-related disorders and methods for treating same |
RU2022107926A (ru) * | 2016-07-05 | 2022-04-27 | Биомарин Текнолоджис Б.В. | Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний |
JOP20190104A1 (ar) * | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
IL297528A (en) | 2016-12-19 | 2022-12-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer conjugates for muscular dystrophy |
EP4406601A2 (en) | 2016-12-19 | 2024-07-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
PT3554553T (pt) | 2016-12-19 | 2022-08-04 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
EP3565577A4 (en) | 2017-01-06 | 2020-10-07 | Avidity Biosciences, Inc. | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCTION OF EXON SKIP |
CN111050806A (zh) | 2017-06-02 | 2020-04-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
JP2020524485A (ja) | 2017-06-02 | 2020-08-20 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | オリゴヌクレオチド組成物及びその使用方法 |
AU2018302098A1 (en) | 2017-07-18 | 2020-02-20 | Csl Behring Gene Therapy, Inc. | Compositions and methods for treating beta-hemoglobinopathies |
GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
US11999953B2 (en) * | 2017-09-13 | 2024-06-04 | The Children's Medical Center Corporation | Compositions and methods for treating transposon associated diseases |
EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
US20200248178A1 (en) | 2017-09-28 | 2020-08-06 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
US11180541B2 (en) * | 2017-09-28 | 2021-11-23 | Geltor, Inc. | Recombinant collagen and elastin molecules and uses thereof |
CN111542336A (zh) | 2017-10-13 | 2020-08-14 | 西莱克塔生物科技公司 | 用于减弱抗病毒转移载体igm应答的方法和组合物 |
JP7394753B2 (ja) | 2017-10-18 | 2023-12-08 | サレプタ セラピューティクス, インコーポレイテッド | アンチセンスオリゴマー化合物 |
KR102443358B1 (ko) | 2017-12-06 | 2022-09-14 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
AU2019252680A1 (en) * | 2018-04-12 | 2020-10-22 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
EP3784248A4 (en) * | 2018-04-26 | 2022-08-10 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATE FOR MUSCLE DYSTROPHY |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
US20220251551A1 (en) * | 2018-06-13 | 2022-08-11 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
JP2021526807A (ja) * | 2018-06-14 | 2021-10-11 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
CN112399849A (zh) | 2018-06-26 | 2021-02-23 | 日本新药株式会社 | 含有反义寡核苷酸的组合物及其在迪谢内肌营养不良症的治疗中的用途 |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
JP2021534799A (ja) * | 2018-08-27 | 2021-12-16 | ノース カロライナ ステート ユニバーシティNorth Carolina State University | 肥満細胞のアポトーシスを誘導するためにスプライススイッチングオリゴヌクレオチドを用いてKitを標的とする方法 |
CA3118167A1 (en) * | 2018-11-02 | 2020-05-07 | Biomarin Technologies B.V. | Bispecific antisense oligonucleotides for dystrophin exon skipping |
BR112021012318A2 (pt) | 2018-12-23 | 2022-01-18 | Csl Behring Llc | Células t doadoras com interruptor de eliminação |
BR112021012240A2 (pt) | 2018-12-23 | 2022-01-18 | Csl Behring Llc | Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich |
EP3918069A1 (en) | 2019-01-31 | 2021-12-08 | Bar Ilan University | Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy |
MX2019001389A (es) | 2019-01-31 | 2020-02-20 | Atso Corp Affairs S A De C V | Oligonucleotidos antisentido de las glutation s transferasa para el tratamiento del cancer. |
KR20210151930A (ko) | 2019-04-12 | 2021-12-14 | 젤터, 인코포레이티드 | 재조합 엘라스틴 및 이의 제조 |
CA3139974A1 (en) | 2019-05-15 | 2020-11-19 | University Of Miami | Treatment of heart disease by disruption of the anchoring of pp2a |
JP2022534741A (ja) | 2019-05-28 | 2022-08-03 | セレクタ バイオサイエンシーズ インコーポレーテッド | 減弱化された抗ウイルス導入ベクター免疫応答のための方法および組成物 |
WO2021092562A1 (en) * | 2019-11-08 | 2021-05-14 | North Carolina State University | Exon skipping of fc-epsilon-ri-beta and ms4a6a in combination for the treatment of allergic diseases |
CA3165316A1 (en) | 2019-12-19 | 2021-06-24 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid enabling exon skipping |
KR20220122673A (ko) | 2019-12-26 | 2022-09-02 | 니뽄 신야쿠 가부시키가이샤 | 엑손 50의 스키핑을 유도하는 안티센스 핵산 |
PE20230237A1 (es) | 2020-02-28 | 2023-02-07 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido que inducen la omision del exon 51 |
US11555190B2 (en) | 2020-03-19 | 2023-01-17 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
US11408000B2 (en) | 2020-06-03 | 2022-08-09 | Triplet Therapeutics, Inc. | Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity |
CN115997015A (zh) | 2020-06-26 | 2023-04-21 | 美国杰特贝林生物制品有限公司 | 具有杀伤开关的供体t细胞 |
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 |
WO2022171972A1 (en) | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
WO2022173811A1 (en) * | 2021-02-12 | 2022-08-18 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting genes associated with cystic fibrosis |
IL309626A (en) | 2021-06-23 | 2024-02-01 | Nippon Shinyaku Co Ltd | A combination of antisense oligomers |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
JPWO2023026994A1 (es) | 2021-08-21 | 2023-03-02 | ||
AU2022345098A1 (en) | 2021-09-16 | 2024-04-04 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
US20230141563A1 (en) | 2021-10-12 | 2023-05-11 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
AU2022424485A1 (en) | 2021-12-27 | 2024-07-11 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic acid compound |
EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
US20230357437A1 (en) | 2022-03-09 | 2023-11-09 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
CN115820642B (zh) * | 2022-11-11 | 2023-10-10 | 昆明理工大学 | 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统 |
US11792125B1 (en) | 2023-05-16 | 2023-10-17 | Citibank, N.A. | Reducing network traffic by filtering network requests based on network request-related information systems and methods |
Family Cites Families (131)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JPS5138722B1 (es) | 1970-12-30 | 1976-10-23 | ||
US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
US5190931A (en) | 1983-10-20 | 1993-03-02 | The Research Foundation Of State University Of New York | Regulation of gene expression by employing translational inhibition of MRNA utilizing interfering complementary MRNA |
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 |
US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
AU5661386A (en) | 1985-03-15 | 1986-10-13 | Stirchak, E. | Stereoregular polynucleotide-binding polymers |
US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
US20020123476A1 (en) | 1991-03-19 | 2002-09-05 | Emanuele R. Martin | Therapeutic delivery compositions and methods of use thereof |
JP2652732B2 (ja) | 1991-09-30 | 1997-09-10 | 三光総業株式会社 | ブロー成型法 |
US5869252A (en) | 1992-03-31 | 1999-02-09 | Abbott Laboratories | Method of multiplex ligase chain reaction |
WO1993020227A1 (en) | 1992-03-31 | 1993-10-14 | Abbott Laboratories | Method of multiplex ligase chain reaction |
US6806084B1 (en) | 1992-06-04 | 2004-10-19 | The Regents Of The University Of California | Methods for compositions for in vivo gene delivery |
JPH07509133A (ja) | 1992-07-17 | 1995-10-12 | リボザイム・ファーマシューティカルズ・インコーポレイテッド | 動物疾患の処置のための方法および剤 |
US5985558A (en) | 1997-04-14 | 1999-11-16 | Isis Pharmaceuticals Inc. | Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos |
EP0698092B1 (en) | 1993-05-11 | 2007-07-25 | The University Of North Carolina At Chapel Hill | Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
US6391636B1 (en) | 1994-05-31 | 2002-05-21 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
GB9510718D0 (en) | 1995-05-26 | 1995-07-19 | Sod Conseils Rech Applic | Antisense oligonucleotides |
WO1997030067A1 (en) | 1996-02-14 | 1997-08-21 | Isis Pharmaceuticals, Inc. | Sugar-modified gapped oligonucleotides |
AU2585197A (en) | 1996-03-20 | 1997-10-10 | Regents Of The University Of California, The | Antisense approach to gene inhibition |
US6153436A (en) | 1997-01-10 | 2000-11-28 | The Board Of Trustees Of The University Of Arkansas | Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions |
US20030114401A1 (en) * | 2001-12-06 | 2003-06-19 | Isis Pharmaceuticals Inc. | Antisense modulation of Ship-1 expression |
GB9819999D0 (en) | 1998-09-14 | 1998-11-04 | Univ London | Treatment of cancer |
US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
DE69905938T2 (de) | 1998-11-13 | 2003-11-13 | Eli Lilly And Co., Indianapolis | Kombination von duloxetin mit nichtsteroid-entzündungshemmern für die behandlung von schmerz |
WO2000045167A2 (en) | 1999-01-29 | 2000-08-03 | Avi Biopharma, Inc. | Non-invasive method for detecting target rna |
US20020049173A1 (en) | 1999-03-26 | 2002-04-25 | Bennett C. Frank | Alteration of cellular behavior by antisense modulation of mRNA processing |
JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
WO2000078341A1 (en) | 1999-06-21 | 2000-12-28 | Murdoch Childrens Research Institute | A method for the prophylaxis and/or treatment of medical disorders |
US7070807B2 (en) | 1999-12-29 | 2006-07-04 | Mixson A James | Branched histidine copolymers and methods for using same |
EP1242052A4 (en) | 1999-12-29 | 2003-07-02 | A James Mixson | HISTIDIN COPOLYMER AND METHODS OF USE THEREOF |
JP2003518946A (ja) | 2000-01-04 | 2003-06-17 | エイブイアイ バイオファーマ, インコーポレイテッド | アンチセンス抗細菌性細胞分裂組成物、およびその方法 |
AU4036600A (en) | 2000-03-28 | 2001-10-08 | Isis Pharmaceuticals Inc | Alteration of cellular behavior by antisense modulation of mrna processing |
US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
ATE437944T1 (de) | 2000-04-28 | 2009-08-15 | Asklepios Biopharmaceutical In | Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung |
AU5736601A (en) | 2000-05-01 | 2001-11-12 | Hybridon Inc | Modulation of oligonucleotide cpg-mediated immune stimulation by positional modification of nucleosides |
AU5752601A (en) | 2000-05-04 | 2001-11-12 | Avi Biopharma Inc | Splice-region antisense composition and method |
JP4836366B2 (ja) | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
AU8696601A (en) | 2000-08-30 | 2002-03-13 | Avi Biopharma Inc | Method for analysis of oligonucleotide analogs |
US6559279B1 (en) | 2000-09-08 | 2003-05-06 | Isis Pharmaceuticals, Inc. | Process for preparing peptide derivatized oligomeric compounds |
US20070037165A1 (en) | 2000-09-08 | 2007-02-15 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
JP2004509619A (ja) | 2000-09-20 | 2004-04-02 | アイシス・ファーマシューティカルス・インコーポレーテッド | Flip−c発現のアンチセンスモジュレーション |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
US6689615B1 (en) | 2000-10-04 | 2004-02-10 | James Murto | Methods and devices for processing blood samples |
JP3781687B2 (ja) | 2001-02-23 | 2006-05-31 | 松下電器産業株式会社 | 遺伝子診断装置及び遺伝子診断方法 |
US6656732B1 (en) | 2001-05-18 | 2003-12-02 | Isis Pharmaceuticals, Inc. | Antisense inhibition of src-c expression |
US20030224353A1 (en) | 2001-10-16 | 2003-12-04 | Stein David A. | Antisense antiviral agent and method for treating ssRNA viral infection |
US7655785B1 (en) | 2002-11-14 | 2010-02-02 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof |
US8090542B2 (en) | 2002-11-14 | 2012-01-03 | Dharmacon Inc. | Functional and hyperfunctional siRNA |
US7314750B2 (en) | 2002-11-20 | 2008-01-01 | Affymetrix, Inc. | Addressable oligonucleotide array of the rat genome |
US7250289B2 (en) | 2002-11-20 | 2007-07-31 | Affymetrix, Inc. | Methods of genetic analysis of mouse |
AU2003295974A1 (en) | 2002-11-25 | 2004-06-18 | Telesector Resources Group, Inc. | Methods and systems for notification of call to device |
ES2561851T3 (es) | 2002-11-25 | 2016-03-01 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
WO2004083432A1 (en) | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
DE602004028930D1 (de) | 2003-04-29 | 2010-10-14 | Avi Biopharma Inc | Zusammensetzungen zur verbesserung der antisense-wirksamkeit und des transports von nukleinsäureanalog in zellen |
EP1495769B1 (en) | 2003-07-11 | 2008-02-27 | LBR Medbiotech B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
KR101032853B1 (ko) | 2003-08-05 | 2011-05-06 | 에이브이아이 바이오파마 인코포레이티드 | 웨스트 나일 바이러스 감염을 치료하기 위한 약학적 조성물 |
US20050048495A1 (en) | 2003-08-29 | 2005-03-03 | Baker Brenda F. | Isoform-specific targeting of splice variants |
AU2004273828A1 (en) | 2003-09-12 | 2005-03-31 | Avi Biopharma, Inc. | Compound and method for treating androgen-independent prostate cancer |
US20050171044A1 (en) | 2003-12-24 | 2005-08-04 | Stein David A. | Oligonucleotide compound and method for treating nidovirus infections |
US20050288246A1 (en) | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
ES2361325T3 (es) | 2004-06-28 | 2011-06-16 | The University Of Western Australia | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
FR2873294B1 (fr) | 2004-07-26 | 2008-05-09 | Greenpharma Sa Sa | Association de medicaments |
FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
US8129352B2 (en) | 2004-09-16 | 2012-03-06 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating ssRNA viral infection |
US20060148740A1 (en) | 2005-01-05 | 2006-07-06 | Prosensa B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
US20120122801A1 (en) | 2005-01-05 | 2012-05-17 | Prosensa B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
JP2008538500A (ja) | 2005-04-22 | 2008-10-30 | アカデミス ツィーケンホイス ライデン | SRタンパク質の結合に対する干渉とRNA二次構造に対する干渉による、mRNA前駆体におけるエクソン認識の調節 |
US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
US8501703B2 (en) | 2005-08-30 | 2013-08-06 | Isis Pharmaceuticals, Inc. | Chimeric oligomeric compounds for modulation of splicing |
US8524676B2 (en) | 2005-09-08 | 2013-09-03 | Sarepta Therapeutics, Inc. | Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds |
US8501704B2 (en) | 2005-11-08 | 2013-08-06 | Sarepta Therapeutics, Inc. | Immunosuppression compound and treatment method |
MX2008006089A (es) | 2005-11-10 | 2009-05-28 | Santaris Pharma As | Oligomeros de conmutacion de empalme para los receptores de la super-familia de tnf y su uso en el tratamiento de enfermedades. |
US20090011004A1 (en) | 2005-12-30 | 2009-01-08 | Philadelphia Health & Education Corp., D/B/A/ Drexel University Of College Of Medicine | Improved carriers for delivery of nucleic acid agents to cells and tissues |
HUE036995T2 (hu) | 2006-05-10 | 2018-08-28 | Sarepta Therapeutics Inc | Az alegységek között kationos kötéssel rendelkezõ oligonukleotid analógok |
US8785407B2 (en) | 2006-05-10 | 2014-07-22 | Sarepta Therapeutics, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
US20070265215A1 (en) | 2006-05-11 | 2007-11-15 | Iversen Patrick L | Antisense restenosis composition and method |
EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
WO2009005793A2 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
WO2009101399A1 (en) | 2008-02-12 | 2009-08-20 | Isis Innovation Limited | Treatment of muscular dystrophy using peptide nucleic acid ( pna) |
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 |
US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
PL3133160T3 (pl) | 2008-10-24 | 2019-06-28 | Sarepta Therapeutics, Inc. | Kompozycje do pomijania eksonów w DMD |
ES2562658T3 (es) | 2008-10-27 | 2016-03-07 | Biomarin Technologies B.V. | Procedimientos y medios para el salto eficiente del exón 45 en el pre-ARNm de la distrofia muscular de Duchenne |
US8592386B2 (en) | 2008-12-17 | 2013-11-26 | Sarepta Therapeutics, Inc. | Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis |
WO2010115993A1 (en) | 2009-04-10 | 2010-10-14 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
US20120046342A1 (en) | 2009-04-24 | 2012-02-23 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
EP2258863A1 (en) | 2009-05-25 | 2010-12-08 | Universita'Degli Studi di Roma "La Sapienza" | miRNA biomarkers for the diagnosis of duchenne muscular dystrophy progression, for monitoring therapeutic interventions, and as therapeutics |
US9034838B2 (en) | 2009-05-25 | 2015-05-19 | Universita Degli Studi Di Roma “La Sapienza” | miR-31 in duchenne muscular dystrophy therapy |
US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
ITTO20090487A1 (it) | 2009-06-26 | 2010-12-27 | Univ Ferrara | Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) |
EP2473607A2 (en) | 2009-08-31 | 2012-07-11 | INSERM - Institut National de la Santé et de la Recherche Médicale | Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy |
IT1397011B1 (it) | 2009-10-14 | 2012-12-20 | Univ Ferrara | Nanoparticella del tipo core-shell idonea per la veicolazione di oligonucleotidi terapeutici in tessuti bersaglio e suo impiego per la preparazione di un medicamento per il trattamento della distrofia muscolare di duchenne. |
KR102239374B1 (ko) | 2009-11-12 | 2021-04-14 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
AU2010319314C1 (en) | 2009-11-13 | 2016-09-01 | Sarepta Therapeutics, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
US9050373B2 (en) | 2010-05-13 | 2015-06-09 | The Charlotte-Mecklenburg Hospital Authority | Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same |
JP2013530154A (ja) | 2010-05-28 | 2013-07-25 | サレプタ セラピューティクス, インコーポレイテッド | 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ |
CN103096928A (zh) | 2010-06-28 | 2013-05-08 | 萩原正敏 | 遗传性疾病的预防/改善剂 |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
SI2623507T1 (sl) | 2010-09-30 | 2017-04-26 | Nippon Shinyaku Co.,Ltd. | Derivati morfolino-nukleinske kisline |
EP2672977A1 (en) | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense oligonucleotides |
JP2012226016A (ja) | 2011-04-15 | 2012-11-15 | Toyota Industries Corp | センサ用光ファイバ |
US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
KR102339196B1 (ko) | 2011-05-05 | 2021-12-15 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
WO2013033407A2 (en) | 2011-08-30 | 2013-03-07 | The Regents Of The University Of California | Identification of small molecules that enhance therapeutic exon skipping |
US20140080896A1 (en) | 2011-08-30 | 2014-03-20 | The Regents Of The University Of California | Identification of small molecules that facilitate therapeutic exon skipping |
DK2581448T3 (en) | 2011-10-13 | 2015-04-27 | Ass Inst De Myologie | Tricyclo-DNA phosphorothioate |
IN2014DN06220A (es) | 2011-12-28 | 2015-10-23 | Nippon Shinyaku Co Ltd | |
JP2015509922A (ja) | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
DE102012203443A1 (de) | 2012-03-05 | 2013-09-05 | Robert Bosch Gmbh | Verfahren zum Bilden eines Kontakts auf einem Halbleitersubstrat und Halbleitervorrichtung |
NZ700399A (en) | 2012-03-20 | 2016-07-29 | Sarepta Therapeutics Inc | Boronic acid conjugates of oligonucleotide analogues |
EP3608407A1 (en) | 2012-07-03 | 2020-02-12 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
KR20150099804A (ko) | 2012-12-20 | 2015-09-01 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물 |
JP6449231B2 (ja) | 2013-03-14 | 2019-01-09 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
PL2970964T3 (pl) | 2013-03-14 | 2019-06-28 | Sarepta Therapeutics, Inc. | Kompozycje pomijające eksony do leczenia dystrofii mięśniowej |
KR20200139271A (ko) | 2013-03-15 | 2020-12-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 개선된 조성물 |
WO2014172660A1 (en) | 2013-04-18 | 2014-10-23 | Brigham Young University | Porous material for thermal and/or electrical isolation and methods of manufacture |
US9862945B2 (en) | 2013-04-20 | 2018-01-09 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs |
EP3355892A4 (en) | 2015-09-30 | 2019-05-22 | Sarepta Therapeutics, Inc. | METHODS OF TREATING MUSCLE DYSTROPHY |
US20200195590A1 (en) | 2018-12-18 | 2020-06-18 | Zipwhip, Inc. | System and method for generating and transmitting automatic reply messages |
-
2005
- 2005-06-28 ES ES05754344T patent/ES2361325T3/es active Active
- 2005-06-28 EP EP10004274.6A patent/EP2206781B1/en not_active Revoked
- 2005-06-28 EP EP12162995.0A patent/EP2500430B1/en active Active
- 2005-06-28 AT AT05754344T patent/ATE498685T1/de active
- 2005-06-28 HU HUE10004274A patent/HUE028632T2/en unknown
- 2005-06-28 PL PL10004274T patent/PL2206781T3/pl unknown
- 2005-06-28 EP EP17159328.8A patent/EP3228711A1/en not_active Withdrawn
- 2005-06-28 PT PT05754344T patent/PT1766010E/pt unknown
- 2005-06-28 ES ES10004274.6T patent/ES2564185T3/es active Active
- 2005-06-28 EP EP20050754344 patent/EP1766010B1/en active Active
- 2005-06-28 DE DE200560026386 patent/DE602005026386D1/de active Active
- 2005-06-28 EP EP20200874.4A patent/EP3808845A1/en not_active Withdrawn
- 2005-06-28 PL PL05754344T patent/PL1766010T3/pl unknown
- 2005-06-28 WO PCT/AU2005/000943 patent/WO2006000057A1/en active Application Filing
- 2005-06-28 EP EP23180290.1A patent/EP4272748A3/en active Pending
- 2005-06-28 SI SI200532044A patent/SI2206781T1/sl unknown
- 2005-06-28 EP EP22152770.8A patent/EP4047096A1/en not_active Withdrawn
- 2005-06-28 US US11/570,691 patent/US7807816B2/en not_active Ceased
- 2005-06-28 DK DK05754344T patent/DK1766010T3/da active
- 2005-06-28 ES ES12162995.0T patent/ES2627917T3/es active Active
- 2005-06-28 DK DK10004274.6T patent/DK2206781T3/en active
- 2005-06-28 EP EP15190341.6A patent/EP3029142A1/en not_active Withdrawn
- 2005-06-28 US US15/349,535 patent/USRE47769E1/en active Active
- 2005-06-28 EP EP15168694.6A patent/EP2933332A1/en not_active Withdrawn
- 2005-06-28 SI SI200531282T patent/SI1766010T1/sl unknown
-
2010
- 2010-07-15 US US12/837,356 patent/US8524880B2/en not_active Ceased
- 2010-07-15 US US12/837,359 patent/US8232384B2/en active Active
- 2010-08-20 US US12/860,078 patent/US7960541B2/en not_active Expired - Fee Related
-
2011
- 2011-05-10 CY CY20111100450T patent/CY1111447T1/el unknown
- 2011-05-12 HR HR20110352T patent/HRP20110352T1/hr unknown
- 2011-06-24 US US13/168,857 patent/US20110263686A1/en not_active Abandoned
- 2011-06-24 US US13/168,863 patent/US8455634B2/en active Active
- 2011-10-11 US US13/270,500 patent/US8450474B2/en active Active
- 2011-10-11 US US13/270,937 patent/US8455635B2/en active Active
- 2011-10-11 US US13/271,080 patent/US8455636B2/en not_active Ceased
- 2011-10-11 US US13/270,744 patent/US8476423B2/en active Active
- 2011-10-11 US US13/270,992 patent/US8486907B2/en not_active Ceased
- 2011-10-11 US US13/270,531 patent/US20120029058A1/en not_active Abandoned
-
2012
- 2012-12-26 US US13/727,415 patent/US20130116310A1/en not_active Abandoned
-
2013
- 2013-01-14 US US13/741,150 patent/US20130253033A1/en not_active Abandoned
- 2013-03-14 US US13/826,880 patent/US9175286B2/en active Active
- 2013-03-14 US US13/826,613 patent/US20130217755A1/en not_active Abandoned
- 2013-05-24 US US13/902,376 patent/US20130253180A1/en not_active Abandoned
- 2013-08-09 US US13/963,578 patent/US20130331438A1/en not_active Abandoned
- 2013-11-21 US US14/086,859 patent/US20140080898A1/en not_active Abandoned
-
2014
- 2014-02-11 US US14/178,059 patent/US20140155587A1/en not_active Abandoned
- 2014-03-24 US US14/223,634 patent/US20150057330A1/en not_active Abandoned
- 2014-05-08 US US14/273,318 patent/US20140243515A1/en not_active Abandoned
- 2014-05-08 US US14/273,379 patent/US20140243516A1/en not_active Abandoned
- 2014-06-26 US US14/316,609 patent/US9024007B2/en active Active
- 2014-06-26 US US14/316,603 patent/US9018368B2/en active Active
- 2014-06-27 US US14/317,952 patent/US9035040B2/en active Active
-
2015
- 2015-06-15 US US14/740,097 patent/US9605262B2/en active Active
- 2015-09-11 US US14/852,149 patent/US9249416B2/en active Active
- 2015-09-11 US US14/852,090 patent/US9441229B2/en active Active
- 2015-09-17 US US14/857,561 patent/US9447415B2/en active Active
- 2015-09-17 US US14/857,555 patent/US9422555B2/en active Active
- 2015-09-18 US US14/858,250 patent/US20160002635A1/en not_active Abandoned
-
2016
- 2016-03-01 HR HRP20160225TT patent/HRP20160225T1/hr unknown
- 2016-03-01 CY CY20161100167T patent/CY1117475T1/el unknown
- 2016-04-20 HK HK16104527.6A patent/HK1216545A1/zh unknown
- 2016-09-23 US US15/274,719 patent/US20170009233A1/en not_active Abandoned
- 2016-09-23 US US15/274,772 patent/US20170009234A1/en not_active Abandoned
-
2017
- 2017-03-23 US US15/467,443 patent/US20180051282A1/en not_active Abandoned
- 2017-07-10 US US15/645,842 patent/USRE47691E1/en active Active
- 2017-07-20 US US15/655,646 patent/USRE47751E1/en active Active
- 2017-08-09 US US15/673,019 patent/US20180163205A1/en not_active Abandoned
- 2017-09-14 US US15/705,172 patent/US9994851B2/en active Active
-
2018
- 2018-04-04 HK HK18104525.6A patent/HK1245325A1/zh unknown
- 2018-08-24 US US16/112,453 patent/US10266827B2/en active Active
- 2018-08-24 US US16/112,371 patent/US10227590B2/en active Active
-
2019
- 2019-01-22 US US16/254,047 patent/US10421966B2/en active Active
- 2019-07-01 US US16/458,929 patent/US10781451B2/en active Active
- 2019-07-31 US US16/527,886 patent/US10968450B2/en active Active
-
2020
- 2020-05-22 US US16/881,430 patent/US10995337B2/en active Active
- 2020-08-19 US US16/997,022 patent/US20210198666A1/en not_active Abandoned
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
ES2564185T3 (es) | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos | |
CN107406396B (zh) | 阳离子性脂质 | |
KR102198736B1 (ko) | 생체 내 약물 전달을 위한 지질 나노입자 및 이의 용도 | |
AU2003266014B2 (en) | Methods for delivery of nucleic acids | |
EP1019365B1 (en) | Novel compositions for the delivery of negatively charged molecules | |
ES2251134T3 (es) | Uso de complejos entre liposomas cationicos y polidesoxirribonucleotidos como medicamentos. | |
CN114727964A (zh) | 脂质纳米粒子的冻干组合物 | |
US10905777B2 (en) | Systems and methods for nucleic acid expression in vivo | |
JP5382682B2 (ja) | 薬物送達複合体 | |
WO2022037465A1 (zh) | 一种脂质纳米颗粒 | |
CN114409554A (zh) | 一种新型阳离子脂质化合物及其组合物和用途 | |
KR20220092363A (ko) | 지질 나노입자를 포함하는 암 예방 또는 치료용 조성물 | |
ES2542864T3 (es) | Composición portadora de administración rápida de ácidos nucleicos | |
JP5931212B2 (ja) | 弱酸性pH応答性ペプチド及び該ペプチドを含むリポソーム | |
Neuberg et al. | Design and evaluation of ionizable peptide amphiphiles for siRNA delivery | |
US20100305190A1 (en) | Nucleic acid complex and nucleic acid delivery composition | |
WO2021193397A1 (ja) | シスチン骨格を有する新規カチオン性脂質 | |
ES2698565A1 (es) | Procedimiento para la elaboración de nanopartículas lipídicas, y nanopartículas lipídicas con los macrófagos cerebrales como células diana | |
EP4268851A1 (en) | Composition for preventing or treating cancer, containing lipid nanoparticles | |
CN118284407A (zh) | 用于将核酸递送至脑组织的脂质纳米颗粒 |