公开(公告)号：US09657312B2

公开(公告)日：2017-05-23

申请号：US14384687

申请日：2012-03-13

申请人： Yeon Soo Kim ,  Moon Kyung Kang ,  A Young Song

发明人： Yeon Soo Kim ,  Moon Kyung Kang ,  A Young Song

IPC分类号： C12N15/86 ,  A61K35/76 ,  A61K38/16 ,  A61K38/45 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K35/76 ,  A61K38/162 ,  A61K38/45 ,  A61K48/00 ,  C12N2740/10032 ,  C12N2740/10043 ,  C12N2740/13045 ,  C12N2740/13052 ,  C12N2800/40 ,  C12N2800/50 ,  C12Y207/01021

摘要： The present invention provides a vector system in which a MuLV-Gag gene, a MuLV-Pol gene, and a GaLV-Env gene are expressed in two separate vectors. The vector system is capable of inserting a therapeutic gene to these two separate vectors, and in this raged, the size of an inserted gene is not limited and a variety of foreign therapeutic genes may be inserted to the vectors. Accordingly, the foreign therapeutic gene may be delivered in a safe and efficient manner to desired tissue of cells of aberrant proliferation. Therefore, the vector system is applicable in a composition for delivering a gene targeting the aberrantly dividing cells of aberrant proliferation, wherein the composition includes a retrovirus produced by cell line transfection. The vector system is also applicable in a composition for preventing or treating a disease caused by cells of aberrant proliferation of, such as cancer cells.

公开(公告)号：US09206440B2

公开(公告)日：2015-12-08

申请号：US13145488

申请日：2010-01-22

申请人： Wen Yang ,  Richard P. Junghans ,  Anthony J. Bais

发明人： Wen Yang ,  Richard P. Junghans ,  Anthony J. Bais

IPC分类号： A01N63/00 ,  C07K16/00 ,  C12P21/08 ,  G01N33/00 ,  C12N5/071 ,  C12N15/86 ,  A61K35/00

CPC分类号： C07K16/18 ,  A61K35/00 ,  A61K38/00 ,  A61K48/00 ,  C07K14/7051 ,  C07K14/70521 ,  C07K16/084 ,  C07K16/40 ,  C07K2317/622 ,  C07K2319/70 ,  C12N7/00 ,  C12N15/86 ,  C12N2740/10032 ,  C12N2740/10052 ,  C12N2740/13043 ,  C12N2740/13045 ,  C12N2810/855 ,  C12N2810/859

摘要： The present invention relates to compositions and methods for creation of vector nucleic acid sequences (e.g., retroviral nucleic acid sequences) that comprise two or more exogenous nucleic acid sequences that encode highly homologous (e.g., identical) polypeptide sequences, yet wherein at least one of the exogenous nucleic acid sequences has been mutated using degenerate codons for purpose of reducing homology between the two or more exogenous nucleic acid sequences while maintaining the encoded polypeptide sequence. Preferred nucleic acid sequences include those encoding multi-chimeric immune receptor (CIR) genes. Specific nucleic acid sequences of such CIR genes are also disclosed.

摘要翻译： 本发明涉及用于产生载体核酸序列（例如逆转录病毒核酸序列）的组合物和方法，其包含编码高度同源（例如相同）多肽序列的两个或多个外源核酸序列，其中至少一个 使用简并密码子突变外源核酸序列，目的是减少两个或多个外源核酸序列之间的同源性，同时保持编码的多肽序列。 优选的核酸序列包括编码多嵌合免疫受体（CIR）基因的那些。 还公开了这种CIR基因的特异性核酸序列。

公开(公告)号：US11739329B2

公开(公告)日：2023-08-29

申请号：US17404576

申请日：2021-08-17

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

CPC分类号： C12N15/1135 ,  A61K35/28 ,  C12N7/00 ,  C12N15/113 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2310/531 ,  C12N2740/10032 ,  C12N2740/10043 ,  C12N2310/14 ,  C12N2310/531

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.

公开(公告)号：US20040265288A1

公开(公告)日：2004-12-30

申请号：US10893199

申请日：2004-07-16

发明人： Michael Z. Gilman

IPC分类号： A61K048/00 ,  C12N005/08

CPC分类号： A61K38/215 ,  A61K38/179 ,  A61K38/1891 ,  A61K38/2257 ,  A61K38/39 ,  A61K38/484 ,  A61K48/005 ,  C07K2319/71 ,  C07K2319/80 ,  C12N2710/10232 ,  C12N2710/16632 ,  C12N2740/10032 ,  C12N2750/14132 ,  C12N2830/00

摘要： This invention provides new applications of regulated gene therapy relating to treatment of cancer and other disorders.

摘要翻译： 本发明提供了与治疗癌症和其他疾病有关的受调节基因治疗的新应用。

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.

公开(公告)号：US20150176026A1

公开(公告)日：2015-06-25

申请号：US14384687

申请日：2012-03-13

申请人： Inje University Industry-Academic Cooperation Foundation

发明人： Yeon Soo Kim ,  Moon Kyung Kang ,  A Young Song

IPC分类号： C12N15/86 ,  A61K38/16 ,  A61K38/45 ,  A61K35/76

CPC分类号： C12N15/86 ,  A61K35/76 ,  A61K38/162 ,  A61K38/45 ,  A61K48/00 ,  C12N2740/10032 ,  C12N2740/10043 ,  C12N2740/13045 ,  C12N2740/13052 ,  C12N2800/40 ,  C12N2800/50 ,  C12Y207/01021

摘要： The present invention provides a vector system in which a MuLV-Gag gene, a MuLV-Pol gene, and a GaLV-Env gene are expressed in two separate vectors. The vector system is capable of inserting a therapeutic gene to these two separate vectors, and in this raged, the size of an inserted gene is not limited and a variety of foreign therapeutic genes may be inserted to the vectors. Accordingly, the foreign therapeutic gene may be delivered in a safe and efficient manner to desired tissue of cells of aberrant proliferation. Therefore, the vector system is applicable in a composition for delivering a gene targeting the aberrantly dividing cells of aberrant proliferation, wherein the composition includes a retrovirus produced by cell line transfection. The vector system is also applicable in a composition for preventing or treating a disease caused by cells of aberrant proliferation of, such as cancer cells.

摘要翻译： 本发明提供了在两个分开的载体中表达MuLV-Gag基因，MuLV-Pol基因和GaLV-Env基因的载体系统。 载体系统能够将治疗基因插入到这两个单独的载体中，并且在这种情况下，插入的基因的大小不受限制，并且可以将多种外来治疗基因插入到载体中。 因此，外来治疗基因可以以安全有效的方式递送到期望的异常增殖细胞组织。 因此，载体系统可用于递送针对异常增殖异常分化细胞的基因的组合物，其中该组合物包含通过细胞系转染产生的逆转录病毒。 载体系统也可用于预防或治疗由诸如癌细胞异常增殖的细胞引起的疾病的组合物。

公开(公告)号：US20120134970A1

公开(公告)日：2012-05-31

申请号：US13145488

申请日：2010-01-22

申请人： Wen Yang ,  Richard P. Junghans ,  Anthony J. Bais

发明人： Wen Yang ,  Richard P. Junghans ,  Anthony J. Bais

IPC分类号： A61K35/76 ,  A61P35/00 ,  C12N5/10 ,  A61P31/12 ,  A61P31/18 ,  A61K35/14 ,  C12N15/63 ,  A61P31/00

CPC分类号： C07K16/18 ,  A61K35/00 ,  A61K38/00 ,  A61K48/00 ,  C07K14/7051 ,  C07K14/70521 ,  C07K16/084 ,  C07K16/40 ,  C07K2317/622 ,  C07K2319/70 ,  C12N7/00 ,  C12N15/86 ,  C12N2740/10032 ,  C12N2740/10052 ,  C12N2740/13043 ,  C12N2740/13045 ,  C12N2810/855 ,  C12N2810/859

摘要： The present invention relates to compositions and methods for creation of vector nucleic acid sequences (e.g., retroviral nucleic acid sequences) that comprise two or more exogenous nucleic acid sequences that encode highly homologous (e.g., identical) polypeptide sequences, yet wherein at least one of the exogenous nucleic acid sequences has been mutated using degenerate codons for purpose of reducing homology between the two or more exogenous nucleic acid sequences while maintaining the encoded polypeptide sequence. Preferred nucleic acid sequences include those encoding multi-chimeric immune receptor (CIR) genes. Specific nucleic acid sequences of such CIR genes are also disclosed.

摘要翻译： 本发明涉及用于产生载体核酸序列（例如逆转录病毒核酸序列）的组合物和方法，其包含编码高度同源（例如相同）多肽序列的两个或多个外源核酸序列，其中至少一个 使用简并密码子突变外源核酸序列，目的是减少两个或多个外源核酸序列之间的同源性，同时保持编码的多肽序列。 优选的核酸序列包括编码多嵌合免疫受体（CIR）基因的那些。 还公开了这种CIR基因的特异性核酸序列。