HK1243727B - 用於治療血紅蛋白病的bcl11a調節 - Google Patents
用於治療血紅蛋白病的bcl11a調節Info
- Publication number
- HK1243727B HK1243727B HK18102648.2A HK18102648A HK1243727B HK 1243727 B HK1243727 B HK 1243727B HK 18102648 A HK18102648 A HK 18102648A HK 1243727 B HK1243727 B HK 1243727B
- Authority
- HK
- Hong Kong
- Prior art keywords
- bcl11a
- hemoglobinopathies
- modulation
- treatment
- Prior art date
Links
- 208000034737 hemoglobinopathy Diseases 0.000 title 1
- 208000018337 inherited hemoglobinopathy Diseases 0.000 title 1
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- 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
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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- Animal Behavior & Ethology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
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Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US9701708P | 2008-09-15 | 2008-09-15 | |
US22257109P | 2009-07-02 | 2009-07-02 |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1243727B true HK1243727B (zh) | 2020-01-10 |
Family
ID=42005791
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK18102648.2A HK1243727B (zh) | 2008-09-15 | 2018-02-23 | 用於治療血紅蛋白病的bcl11a調節 |
Country Status (11)
Country | Link |
---|---|
US (5) | US8383604B2 (zh) |
EP (2) | EP2334794B8 (zh) |
BR (1) | BRPI0919017A2 (zh) |
CA (1) | CA2737180C (zh) |
DK (1) | DK2334794T3 (zh) |
ES (2) | ES2615829T3 (zh) |
HK (1) | HK1243727B (zh) |
MX (2) | MX345116B (zh) |
PT (1) | PT2334794T (zh) |
WO (1) | WO2010030963A2 (zh) |
ZA (1) | ZA201101964B (zh) |
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US20140271657A1 (en) * | 2011-10-12 | 2014-09-18 | 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 |
ES2641840T3 (es) | 2012-02-24 | 2017-11-14 | Fred Hutchinson Cancer Research Center | Composiciones y métodos para el tratamiento de hemoglobinopatías |
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KR102243092B1 (ko) | 2012-12-06 | 2021-04-22 | 시그마-알드리치 컴퍼니., 엘엘씨 | Crispr-기초된 유전체 변형과 조절 |
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CN116083487A (zh) | 2013-05-15 | 2023-05-09 | 桑格摩生物治疗股份有限公司 | 用于治疗遗传病状的方法和组合物 |
WO2014188001A1 (en) | 2013-05-24 | 2014-11-27 | Santaris Pharma A/S | Oligonucleotide modulators of b-cell cll/lymphoma 11a (bcl11a) and uses thereof |
CA2915842C (en) | 2013-06-17 | 2022-11-29 | The Broad Institute, Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
KR20160056869A (ko) | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
CA2915845A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
PL3492593T3 (pl) | 2013-11-13 | 2022-04-19 | Children's Medical Center Corporation | Regulacja ekspresji genów, w której pośredniczą nukleazy |
CN106536729A (zh) | 2013-12-12 | 2017-03-22 | 布罗德研究所有限公司 | 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用 |
WO2015089462A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
CA2946309C (en) | 2014-04-25 | 2021-11-09 | Michael MILSOM | Synthetic bcl11a micrornas for treating hemoglobinopathies |
WO2016094867A1 (en) | 2014-12-12 | 2016-06-16 | 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 |
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KR20220145913A (ko) | 2016-08-24 | 2022-10-31 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
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MA50849A (fr) | 2017-10-26 | 2020-09-02 | Vertex Pharma | Substances et procédés pour le traitement d'hémoglobinopathies |
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ZA201101964B (en) | 2012-05-30 |
ES2615829T3 (es) | 2017-06-08 |
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EP2334794A2 (en) | 2011-06-22 |
BRPI0919017A2 (pt) | 2017-05-30 |
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