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1. 发明授权

US11261441B2 Vectors and compositions for treating hemoglobinopathies 有权
公开(公告)号：US11261441B2
公开(公告)日：2022-03-01
申请号：US16496720
申请日：2018-03-29
申请人： bluebird bio, Inc. , CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Gabor Veres , David A. Williams
IPC分类号： C12N15/113 , A61K9/00 , A61K31/7088 , C12N15/86
摘要： The invention provides improved gene therapy vectors, compositions, and methods.

2. 发明申请

US20220259594A1 VECTORS AND COMPOSITIONS FOR TREATING HEMOGLOBINOPATHIES 有权
公开(公告)号：US20220259594A1
公开(公告)日：2022-08-18
申请号：US17587999
申请日：2022-01-28
申请人： bluebird bio, Inc. , Children's Medical Center Corporation
发明人： Gabor Veres , David A. Williams
IPC分类号： C12N15/113 , A61K9/00 , A61K31/7088 , C12N15/86
摘要： The invention provides improved gene therapy vectors, compositions, and methods.

3. 发明授权

US10662429B2 Compositions and methods to treating hemoglobinopathies 有权
公开(公告)号：US10662429B2
公开(公告)日：2020-05-26
申请号：US16366900
申请日：2019-03-27
申请人： THE CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Michael Milsom , David A. Williams , Richard Gregory
IPC分类号： C07H21/04 , C12N15/113 , A61K35/28 , C12N7/00
摘要： Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.

4. 发明授权

US09353166B2 Chimeric peptides for the regulation of GTPases 有权
标题翻译：嵌合肽用于调节GTP酶
公开(公告)号：US09353166B2
公开(公告)日：2016-05-31
申请号：US14216318
申请日：2014-03-17
申请人： Children's Medical Center Corporation
发明人： Yi Zheng , David A. Williams
IPC分类号： C07K14/47 , C12N9/14
CPC分类号： C07K14/4706 , C07K2319/01 , C07K2319/02 , C07K2319/03 , C12N9/14 , C12Y306/05002
摘要： Chimeric peptides or fusion proteins are disclosed that include a RhoGAP activity domain and at least one specificity domain that targets a specific Rho protein. The fusion proteins can be used to inhibit any GTPase activity within a cell. The fusion proteins are particularly advantageous for the treatment of cancer. The present invention generally relates to chimeric peptides capable of regulating GTPases, and more particularly, to methods of targeting individual GTPases by using GTPase-activating proteins. Such proteins may be used for the treatment of cancers and other GTPase-related diseases. This invention relates to nucleic acid molecules and the encoded GTPase activating proteins, and variants thereof, and to the use of these molecules in the characterization, diagnosis, prevention, and treatment of cell signaling, immune, and cell proliferative disorders, particularly cancer. Disclosed herein are compounds and methods for regulating transcription of a selected gene.
摘要翻译：公开了嵌合肽或融合蛋白，其包括RhoGAP活性结构域和至少一个靶向特异性Rho蛋白的特异性结构域。融合蛋白可用于抑制细胞内的任何GTP酶活性。融合蛋白对癌症的治疗特别有利。本发明一般涉及能够调节GTP酶的嵌合肽，更具体地，涉及通过使用GTP酶活化蛋白靶向各种GTP酶的方法。这些蛋白质可用于治疗癌症和其它与GTP酶有关的疾病。本发明涉及核酸分子和编码的GTP酶活化蛋白及其变体，以及这些分子在表征，诊断，预防和治疗细胞信号传导，免疫和细胞增殖性疾病，特别是癌症中的用途。本文公开了用于调节所选基因转录的化合物和方法。

5. 发明申请

US20170218372A1 COMPOSITIONS AND METHODS TO TREATING HEMOGLOBINOPATHIES 审中-公开
公开(公告)号：US20170218372A1
公开(公告)日：2017-08-03
申请号：US15306527
申请日：2015-04-24
申请人： CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Michael Milsom , David A. Williams , Richard Gregory
IPC分类号： C12N15/113 , C12N7/00 , A61K35/28
CPC分类号： C12N15/1135 , A61K35/28 , C12N7/00 , C12N15/113 , C12N2310/141 , C12N2310/3519 , C12N2310/531 , C12N2740/10032 , C12N2740/10043 , C12N2310/14
摘要： Embodiment herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods o use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels. In particular illustrative embodiment, the present invention provides, in part, improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematopoietic-related disorders.

6. 发明申请

US20150265608A1 Compounds for Treating Rac-GTPase Mediated Disorder 审中-公开
标题翻译：用于治疗Rac-GTPase介导的疾病的化合物
公开(公告)号：US20150265608A1
公开(公告)日：2015-09-24
申请号：US14434916
申请日：2013-10-11
申请人： Children's Medical Center Corporation
发明人： David A. Williams
IPC分类号： A61K31/496 , A61K31/4245 , A61K31/4184 , A61K31/42 , A61K31/4162 , A61K31/4439 , A61K31/422
CPC分类号： A61K31/496 , A61K31/4162 , A61K31/4184 , A61K31/42 , A61K31/422 , A61K31/4245 , A61K31/4439 , A61K31/63
摘要： This disclosure relates to compositions including certain compounds identified by a quantitative, high throughput assay to be effective in the treatment of chronic myelogenous leukemia, as well as methods for the manufacture of and the use of these compounds for treating a Rac-GTPase mediated disorder.
摘要翻译：本公开涉及包括通过定量，高通量测定法鉴定的有效治疗慢性骨髓性白血病的某些化合物的组合物，以及用于制备和使用这些化合物用于治疗Rac-GTPase介导的病症的方法。

7. 发明授权

US11124794B2 Compositions and methods to treating hemoglobinopathies 有权
公开(公告)号：US11124794B2
公开(公告)日：2021-09-21
申请号：US16841259
申请日：2020-04-06
申请人： THE CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Michael Milsom , David A. Williams , Richard Gregory
IPC分类号： C07H21/04 , C12N15/113 , A61K35/28 , C12N7/00
摘要： Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.

8. 发明授权

US10287588B2 Compositions and methods to treating hemoglobinopathies 有权
公开(公告)号：US10287588B2
公开(公告)日：2019-05-14
申请号：US15306527
申请日：2015-04-24
申请人： CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Michael Milsom , David A. Williams , Richard Gregory
IPC分类号： C07H21/04 , C12N15/113 , A61K35/28 , C12N7/00
摘要： Embodiment herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels. In particular illustrative embodiment, the present invention provides, in part, improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematopoietic-related disorders.

9. 发明申请

US20150072937A1 Chimeric Peptides for the Regulation of GTPases 有权
标题翻译：嵌合肽用于调节GTP酶
公开(公告)号：US20150072937A1
公开(公告)日：2015-03-12
申请号：US14216318
申请日：2014-03-17
申请人： Children's Medical Center Corporation
发明人： Yi Zheng , David A. Williams
IPC分类号： C07K14/47 , C12N9/14
CPC分类号： C07K14/4706 , C07K2319/01 , C07K2319/02 , C07K2319/03 , C12N9/14 , C12Y306/05002
摘要： Chimeric peptides or fusion proteins are disclosed that include a RhoGAP activity domain and at least one specificity domain that targets a specific Rho protein. The fusion proteins can be used to inhibit any GTPase activity within a cell. The fusion proteins are particularly advantageous for the treatment of cancer. The present invention generally relates to chimeric peptides capable of regulating GTPases, and more particularly, to methods of targeting individual GTPases by using GTPase-activating proteins. Such proteins may be used for the treatment of cancers and other GTPase-related diseases. This invention relates to nucleic acid molecules and the encoded GTPase activating proteins, and variants thereof, and to the use of these molecules in the characterization, diagnosis, prevention, and treatment of cell signaling, immune, and cell proliferative disorders, particularly cancer. Disclosed herein are compounds and methods for regulating transcription of a selected gene.
摘要翻译：公开了嵌合肽或融合蛋白，其包括RhoGAP活性结构域和至少一个靶向特异性Rho蛋白的特异性结构域。融合蛋白可用于抑制细胞内的任何GTP酶活性。融合蛋白对癌症的治疗特别有利。本发明一般涉及能够调节GTP酶的嵌合肽，更具体地，涉及通过使用GTP酶活化蛋白靶向各种GTP酶的方法。这些蛋白质可用于治疗癌症和其它与GTP酶有关的疾病。本发明涉及核酸分子和编码的GTP酶活化蛋白及其变体，以及这些分子在表征，诊断，预防和治疗细胞信号传导，免疫和细胞增殖性疾病，特别是癌症中的用途。本文公开了用于调节所选基因转录的化合物和方法。

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