MX345116B - Modulacion de bcl11a para tratamiento de hemoglobinopatias. - Google Patents
Modulacion de bcl11a para tratamiento de hemoglobinopatias.Info
- Publication number
- MX345116B MX345116B MX2014006638A MX2014006638A MX345116B MX 345116 B MX345116 B MX 345116B MX 2014006638 A MX2014006638 A MX 2014006638A MX 2014006638 A MX2014006638 A MX 2014006638A MX 345116 B MX345116 B MX 345116B
- Authority
- MX
- Mexico
- Prior art keywords
- bcl11a
- hemoglobinopathies
- modulation
- treatment
- expression
- Prior art date
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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/795—Porphyrin- or corrin-ring-containing peptides
- C07K14/805—Haemoglobins; Myoglobins
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0381—Animal model for diseases of the hematopoietic 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
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/60—Transcription factors
-
- 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
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/998—Proteins not provided for elsewhere
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/20—Pseudochromosomes, minichrosomosomes
- C12N2800/206—Pseudochromosomes, minichrosomosomes of yeast origin, e.g. YAC, 2u
Abstract
La presente invención se refiere a una composición que comprende un inhibidor de BCL11A para su uso en el tratamiento y/o prevención de una hemoglobinapatía, caracterizada porque el inhibidor de BCL11A es un ácido nucleico específico de BCL11A que inhibe la expresión de BCL11A, y en donde el ácido nucleico específico de BCL11 es un agente de interferencia de RNA específico de BCL11A, un vector que codifica al agente de interferencia de RNA específico de BCL11A, un constructo o molécula de antisentido o una ribozima.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US9701708P | 2008-09-15 | 2008-09-15 | |
US22257109P | 2009-07-02 | 2009-07-02 | |
PCT/US2009/056770 WO2010030963A2 (en) | 2008-09-15 | 2009-09-14 | Modulation of bcl11a for treatment of hemoglobinopathies |
Publications (1)
Publication Number | Publication Date |
---|---|
MX345116B true MX345116B (es) | 2017-01-17 |
Family
ID=42005791
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2011002731A MX2011002731A (es) | 2008-09-15 | 2009-09-14 | Modulacion de bcl11a para tratamiento de hemoglobinopatias. |
MX2014006638A MX345116B (es) | 2008-09-15 | 2009-09-14 | Modulacion de bcl11a para tratamiento de hemoglobinopatias. |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2011002731A MX2011002731A (es) | 2008-09-15 | 2009-09-14 | Modulacion de bcl11a para tratamiento de hemoglobinopatias. |
Country Status (11)
Country | Link |
---|---|
US (5) | US8383604B2 (es) |
EP (2) | EP3208339B1 (es) |
BR (1) | BRPI0919017A2 (es) |
CA (1) | CA2737180C (es) |
DK (1) | DK2334794T3 (es) |
ES (2) | ES2738980T3 (es) |
HK (1) | HK1243727B (es) |
MX (2) | MX2011002731A (es) |
PT (1) | PT2334794T (es) |
WO (1) | WO2010030963A2 (es) |
ZA (1) | ZA201101964B (es) |
Families Citing this family (59)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3208339B1 (en) | 2008-09-15 | 2019-05-01 | The Children's Medical Center Corporation | Modulation of bcl11a for treatment of hemoglobinopathies |
WO2012073047A2 (en) | 2010-12-03 | 2012-06-07 | Genome Research Limited | Compositions and methods |
EP2648763A4 (en) | 2010-12-10 | 2014-05-14 | Alnylam Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A |
EP2766041B1 (en) * | 2011-10-12 | 2018-12-05 | Children's Medical Center Corporation | Combinatorial compositions and methods of treating hemoglobinopathies |
GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
EP3272356A1 (en) * | 2012-02-24 | 2018-01-24 | Fred Hutchinson Cancer Research Center | Compositions and methods for the treatment of hemoglobinopathies |
LT3401400T (lt) | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
ES2812599T3 (es) | 2012-08-29 | 2021-03-17 | Sangamo Therapeutics Inc | Procedimientos y composiciones para el tratamiento de una afección genética |
PT3502240T (pt) | 2012-11-27 | 2021-08-11 | Childrens Medical Ct Corp | Elementos reguladores distais de bcl11a como alvo para a reindução de hemoglobina fetal |
EP3138911B1 (en) | 2012-12-06 | 2018-12-05 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
ES2658401T3 (es) | 2012-12-12 | 2018-03-09 | The Broad Institute, Inc. | Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas |
KR101780885B1 (ko) | 2013-03-14 | 2017-10-11 | 카리부 바이오사이언시스 인코포레이티드 | 핵산-표적화 핵산의 조성물 및 방법 |
CA2910489A1 (en) * | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
KR20160013110A (ko) | 2013-05-24 | 2016-02-03 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | B-세포 cll/림프종 11a(bcl11a)의 올리고뉴클레오티드 조절제 및 이의 용도 |
DK3011032T3 (da) | 2013-06-17 | 2020-01-20 | Broad Inst Inc | Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til målretning mod og modellering af sygdomme og forstyrrelser i postmitotiske celler |
EP3011034B1 (en) | 2013-06-17 | 2019-08-07 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
KR20160030187A (ko) | 2013-06-17 | 2016-03-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 간의 표적화 및 치료를 위한 CRISPRCas 시스템, 벡터 및 조성물의 전달 및 용도 |
EP3960856A1 (en) | 2013-11-13 | 2022-03-02 | Children's Medical Center Corporation | Nuclease-mediated regulation of gene expression |
KR20160089527A (ko) | 2013-12-12 | 2016-07-27 | 더 브로드 인스티튜트, 인코퍼레이티드 | 게놈 편집을 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료적 응용 |
MX2016007327A (es) | 2013-12-12 | 2017-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para dirigirlos a trastornos y enfermedades usando componentes para suministro de particulas. |
BR112016024565A2 (pt) | 2014-04-25 | 2018-01-23 | Children's Medical Center Corporation | composições e métodos de tratar hemoglobinopatias |
WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
EP3985115A1 (en) | 2014-12-12 | 2022-04-20 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
US20180200387A1 (en) | 2015-02-23 | 2018-07-19 | Crispr Therapeutics Ag | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
EP3262172A2 (en) | 2015-02-23 | 2018-01-03 | Crispr Therapeutics AG | Materials and methods for treatment of hemoglobinopathies |
ES2835861T3 (es) | 2015-05-08 | 2021-06-23 | Childrens Medical Ct Corp | Direccionamiento de regiones funcionales del potenciador de BCL11A para la reinducción de hemoglobina fetal |
US10808020B2 (en) | 2015-05-12 | 2020-10-20 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
WO2016205749A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
WO2017077394A2 (en) | 2015-11-04 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
US20190316154A1 (en) * | 2016-07-07 | 2019-10-17 | St. Jude Children's Research Hospital | Erythroid-specific promoter and method of use thereof |
WO2018039440A1 (en) | 2016-08-24 | 2018-03-01 | Sangamo Therapeutics, Inc. | Regulation of gene expression using engineered nucleases |
AU2017315414B2 (en) | 2016-08-24 | 2024-02-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
US11261441B2 (en) | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
US11788087B2 (en) | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
MA50849A (fr) | 2017-10-26 | 2020-09-02 | Vertex Pharma | Substances et procédés pour le traitement d'hémoglobinopathies |
SG11202011975WA (en) | 2018-06-05 | 2020-12-30 | Lifeedit Inc | Rna-guided nucleases and active fragments and variants thereof and methods of use |
CA3124110A1 (en) | 2018-12-17 | 2020-06-25 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use thereof |
MX2021007835A (es) | 2018-12-27 | 2021-08-11 | Lifeedit Therapeutics Inc | Polipeptidos utiles para edicion de genes y metodos de uso. |
WO2020146758A1 (en) * | 2019-01-10 | 2020-07-16 | The General Hospital Corporation | Methods to treat mitochondrial-associated dysfunctions or diseases |
CA3135759A1 (en) | 2019-04-30 | 2020-11-05 | David Baram | Crispr nuclease |
EP4013864A1 (en) | 2019-08-12 | 2022-06-22 | Lifeedit Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
JP2022550599A (ja) | 2019-10-03 | 2022-12-02 | アーティサン ディベロップメント ラブズ インコーポレイテッド | 操作されたデュアルガイド核酸を有するcrisprシステム |
WO2021138247A1 (en) | 2019-12-30 | 2021-07-08 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
TW202208626A (zh) | 2020-04-24 | 2022-03-01 | 美商生命編輯公司 | Rna引導核酸酶及其活性片段與變體,以及使用方法 |
WO2021231437A1 (en) | 2020-05-11 | 2021-11-18 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleic acid binding proteins and active fragments and variants thereof and methods of use |
WO2022256448A2 (en) | 2021-06-01 | 2022-12-08 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing, or modifying genes |
EP4359527A2 (en) * | 2021-06-23 | 2024-05-01 | Novartis AG | Compositions and methods for the treatment of hemoglobinopathies |
WO2023139557A1 (en) | 2022-01-24 | 2023-07-27 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
USD1016111S1 (en) | 2022-01-28 | 2024-02-27 | Milwaukee Electric Tool Corporation | Strut shearing die |
USD1012142S1 (en) | 2022-01-28 | 2024-01-23 | Milwaukee Electric Tool Corporation | Strut shearing die |
WO2023167882A1 (en) | 2022-03-01 | 2023-09-07 | Artisan Development Labs, Inc. | Composition and methods for transgene insertion |
WO2023215877A1 (en) * | 2022-05-06 | 2023-11-09 | The Children's Medical Center Corporation | Compositions and methods for targeted protein degradation |
EP4306265A3 (en) | 2022-06-17 | 2024-04-24 | Milwaukee Electric Tool Corporation | Shear cutting tool |
WO2024033901A1 (en) | 2022-08-12 | 2024-02-15 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5928638A (en) * | 1996-06-17 | 1999-07-27 | Systemix, Inc. | Methods for gene transfer |
US8101349B2 (en) | 1997-12-23 | 2012-01-24 | Novartis Vaccines And Diagnostics, Inc. | Gene products differentially expressed in cancerous cells and their methods of use II |
JP4095895B2 (ja) * | 2000-12-01 | 2008-06-04 | マックス−プランク−ゲゼルシャフト ツール フォーデルング デル ヴィッセンシャフテン エー.ヴェー. | Rna干渉を媒介する短鎖rna分子 |
EP2314691A3 (en) * | 2002-11-14 | 2012-01-18 | Dharmacon, Inc. | Fuctional and hyperfunctional siRNA |
EP1752536A4 (en) * | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
US20080051431A1 (en) * | 2006-05-26 | 2008-02-28 | Dominique Verhelle | Methods and compositions using immunomodulatory compounds in combination therapy |
EP3031916B1 (en) | 2007-06-11 | 2017-06-07 | Takara Bio Inc. | Method for expression of specific gene |
GB0713183D0 (en) * | 2007-07-06 | 2007-08-15 | King S College London | Method |
EP3208339B1 (en) | 2008-09-15 | 2019-05-01 | The Children's Medical Center Corporation | Modulation of bcl11a for treatment of hemoglobinopathies |
WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
WO2016182893A1 (en) | 2015-05-08 | 2016-11-17 | Teh Broad Institute Inc. | Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof |
-
2009
- 2009-09-14 EP EP16194659.5A patent/EP3208339B1/en active Active
- 2009-09-14 BR BRPI0919017A patent/BRPI0919017A2/pt not_active Application Discontinuation
- 2009-09-14 MX MX2011002731A patent/MX2011002731A/es active IP Right Grant
- 2009-09-14 ES ES16194659T patent/ES2738980T3/es active Active
- 2009-09-14 WO PCT/US2009/056770 patent/WO2010030963A2/en active Application Filing
- 2009-09-14 EP EP09813730.0A patent/EP2334794B8/en active Active
- 2009-09-14 CA CA2737180A patent/CA2737180C/en active Active
- 2009-09-14 PT PT98137300T patent/PT2334794T/pt unknown
- 2009-09-14 MX MX2014006638A patent/MX345116B/es unknown
- 2009-09-14 ES ES09813730.0T patent/ES2615829T3/es active Active
- 2009-09-14 US US13/063,524 patent/US8383604B2/en active Active
- 2009-09-14 DK DK09813730.0T patent/DK2334794T3/en active
-
2011
- 2011-03-15 ZA ZA2011/01964A patent/ZA201101964B/en unknown
-
2013
- 2013-01-17 US US13/743,399 patent/US9228185B2/en active Active
-
2016
- 2016-01-04 US US14/987,219 patent/US9885041B2/en active Active
-
2018
- 2018-02-05 US US15/888,350 patent/US10266826B2/en active Active
- 2018-02-23 HK HK18102648.2A patent/HK1243727B/zh unknown
-
2019
- 2019-02-14 US US16/275,534 patent/US10570392B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
US9228185B2 (en) | 2016-01-05 |
PT2334794T (pt) | 2017-02-15 |
CA2737180A1 (en) | 2010-03-18 |
EP2334794B1 (en) | 2016-11-09 |
US20110182867A1 (en) | 2011-07-28 |
EP2334794A4 (en) | 2012-04-18 |
ES2738980T3 (es) | 2020-01-28 |
EP2334794A2 (en) | 2011-06-22 |
US8383604B2 (en) | 2013-02-26 |
DK2334794T3 (en) | 2017-02-20 |
WO2010030963A3 (en) | 2010-07-29 |
US20190169615A1 (en) | 2019-06-06 |
ES2615829T3 (es) | 2017-06-08 |
EP2334794B8 (en) | 2017-04-19 |
US10266826B2 (en) | 2019-04-23 |
US20180179527A1 (en) | 2018-06-28 |
CA2737180C (en) | 2019-02-19 |
MX2011002731A (es) | 2011-04-26 |
US20170152513A1 (en) | 2017-06-01 |
EP3208339A1 (en) | 2017-08-23 |
US20130129629A1 (en) | 2013-05-23 |
WO2010030963A2 (en) | 2010-03-18 |
BRPI0919017A2 (pt) | 2017-05-30 |
US9885041B2 (en) | 2018-02-06 |
HK1243727B (zh) | 2020-01-10 |
WO2010030963A9 (en) | 2010-06-10 |
US10570392B2 (en) | 2020-02-25 |
EP3208339B1 (en) | 2019-05-01 |
ZA201101964B (en) | 2012-05-30 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX345116B (es) | Modulacion de bcl11a para tratamiento de hemoglobinopatias. | |
MX2012009088A (es) | Anticuerpos que se enlazan a lisil oxidasa-tipo2 (loxl2) y metodos de uso para los mismos. | |
SG10201804330YA (en) | Engineered high-affinity human t cell receptors | |
MX2013014544A (es) | Vectorales de terapia genica para adrenoleucodistrofia y adrenomieloneuropatia. | |
IN2014MN01970A (es) | ||
BR112017017810A2 (pt) | materiais e métodos para tratamento de hemoglobinopatias | |
NZ707831A (en) | Agents for treatment of claudin expressing cancer diseases | |
MY191313A (en) | Use of chimeric antigen receptor-modified t cells to treat cancer | |
MX2015003874A (es) | Modulacion de ire1. | |
WO2012062810A3 (en) | Cell culture medium and process for protein expression, said medium and process comprising a pam inhibitor | |
IN2015DN02939A (es) | ||
MX2013003309A (es) | Compuestos de benzamida sustituidos. | |
MX2019007494A (es) | Metodo para modular el crecimiento de celulas madre hematopoyeticas. | |
SG10201804869UA (en) | Purification of biological molecules | |
BR112014031424A2 (pt) | tratamento de células pluripotentes | |
MY159971A (en) | Multipotent/pluripotent cells and methods | |
WO2011159684A3 (en) | Generation of induced pluripotent stem cells from small volumes of peripheral blood | |
PH12014501032A1 (en) | 2-thiopyrimidinones | |
EA201590507A1 (ru) | Композиции и способы, предусматривающие последовательности, определяющие мужскую фертильность | |
WO2012053823A3 (ko) | 인자 ix 활성을 갖는 융합 단백질 | |
MX2014010940A (es) | El uso de antitrombina en el tratamiento de preeclampsia. | |
GB201316955D0 (en) | Composition comprising a diindolymethane and a retinoid to treat a skin condition | |
WO2014012115A3 (en) | Method for inducing cells to less mature state | |
MX2010001187A (es) | Moduladores del receptor ccr9 y metodos para su uso. | |
EP2597949A4 (en) | SUBMIKROMETERZUSAMMENSETZUNGEN |