-
公开(公告)号：US20200316223A1
公开(公告)日：2020-10-08
申请号：US16824008
申请日：2020-03-19
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , Hillard Rubin
摘要： Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
2.发明申请公开(公告)号：US20210130794A1
公开(公告)日：2021-05-06
申请号：US17073862
申请日：2020-10-19
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , James Anthony McSwiggen , Jaime Michelle Prout , Danielle Lauren Sookiasian
IPC分类号： C12N7/00
摘要： Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
公开(公告)号：US20200289674A1
公开(公告)日：2020-09-17
申请号：US16798890
申请日：2020-02-24
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , Hillard Rubin
摘要： Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
4.发明申请公开(公告)号：US20190256867A1
公开(公告)日：2019-08-22
申请号：US16265684
申请日：2019-02-01
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , James Anthony McSwiggen , Jaime Michelle Prout , Danielle Lauren Sookiasian
IPC分类号： C12N15/90 , A61K35/761 , C12N7/00
摘要： Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
公开(公告)号：US11951183B2
公开(公告)日：2024-04-09
申请号：US16824008
申请日：2020-03-19
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , Hillard Rubin
CPC分类号： A61K48/0066 , C12N15/86 , C12N2750/14143 , C12N2750/14152 , C12N2750/14171 , C12N2830/32
摘要： Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
6.发明申请公开(公告)号：US20190134118A1
公开(公告)日：2019-05-09
申请号：US16163061
申请日：2018-10-17
发明人： Saswati Chatterjee , Kamehameha K. Wong, Jr. , Marwa BenHajSalah , Laura Jane Smith , Albert Barnes Seymour , Jason Boke Wright , James Anthony McSwiggen , Serena Nicole Dollive , Thia Baboval St. Martin , Jaime Michelle Prout
IPC分类号： A61K35/761 , C12N15/62 , C07K14/075 , A61K48/00
摘要： Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions.
-
公开(公告)号：US12064486B2
公开(公告)日：2024-08-20
申请号：US16798890
申请日：2020-02-24
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , Hillard Rubin
CPC分类号： A61K48/0066 , C12N15/86 , C12N2750/14143 , C12N2750/14152 , C12N2750/14171 , C12N2830/32
摘要： Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
公开(公告)号：US20230374546A1
公开(公告)日：2023-11-23
申请号：US18182001
申请日：2023-03-10
发明人： Carmen BARNES , Yogeshwar Sharma , Omar Francone , Hillard Rubin , Wei Wang , Gustavo Cerqueira , Serena Nicole Dollive , Andrew Pla
CPC分类号： C12N15/86 , C07K16/18 , A61K48/0066 , A61K48/0041 , A61P37/06 , C12N2750/14021 , C12N2750/14043 , C12N2750/14052 , C12N2750/14071 , C12N2830/50 , C12N2830/008 , A61K2039/505
摘要： Provided herein are polynucleotides comprising novel bidirectional dual expression cassettes, recombination adeno-associated virus (rAAV) comprising these polynucleotides, and methods of making and using the polynucleotides and rAAV. Also provided are novel transcriptional control elements (e.g., promoters, enhancers, introns, polyadenylation sequences, and combinations thereof), and novel antibody coding sequences. The compositions and methods disclosed herein are particularly advantageous in that they allow for the efficient expression of two different polypeptides (e.g., an antibody heavy chain and an antibody light chain) in a cell. In particular, they allow for the efficient expression of antibodies (e.g., anti-C5 antibodies) in a subject, for the treatment of diseases (e.g., C5-mediated diseases, such as PNH).
-
公开(公告)号：US20190231901A1
公开(公告)日：2019-08-01
申请号：US16369879
申请日：2019-03-29
发明人： Albert Barnes Seymour , Seemin Seher Ahmed , Jason Boke Wright , Serena Nicole Dollive , Hillard Rubin
CPC分类号： A61K48/0066 , C12N15/86 , C12N2750/14143 , C12N2750/14152 , C12N2750/14171 , C12N2830/32
摘要： Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
-
-
-
-
-
-
-
-
搜索结果
9 条记录 (耗时 0.015 秒)
