公开(公告)号：US08445267B2

公开(公告)日：2013-05-21

申请号：US12595196

申请日：2008-04-08

Applicant: Li Zhong ,  Sergei Zolotukhin ,  Lakshmanan Govindasamy ,  Mavis Agbandje-McKenna ,  Arun Srivastava

Inventor： Li Zhong ,  Sergei Zolotukhin ,  Lakshmanan Govindasamy ,  Mavis Agbandje-McKenna ,  Arun Srivastava

IPC: C12N15/00 ,  C12N15/09

CPC classification number: C07K14/005 ,  A61K35/76 ,  A61K39/0011 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0091 ,  A61K2039/5158 ,  C12N7/00 ,  C12N15/86 ,  C12N15/8645 ,  C12N2750/14122 ,  C12N2750/14132 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  C12N2810/6027

Abstract: Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.

Abstract translation: 公开了酪氨酸修饰的rAAV载体，以及包含它们的感染性病毒粒子，组合物和药物制剂。 还公开了制备方法和使用所公开的酪氨酸磷酸化衣壳蛋白突变体rAAV载体在多种诊断和治疗应用中的方法，包括体内和离体基因治疗以及大规模生产rAAV载体。

公开(公告)号：US20110294218A1

公开(公告)日：2011-12-01

申请号：US13097046

申请日：2011-04-28

Applicant: Saswati Chatterjee ,  Laura Smith ,  Kamehameha Wong

Inventor： Saswati Chatterjee ,  Laura Smith ,  Kamehameha Wong

IPC: C12N15/85 ,  C07K14/075 ,  C07H21/04

CPC classification number: C07K14/005 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2810/6027

Abstract: Novel adeno-associated virus (AAV) isolates in nucleotide and amino acid forms and uses thereof are provided. The isolates show tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Discrete modified portions of the cap gene, VP1, VP2, and VP3, may be used alone or in combination in the present methods.

Abstract translation: 提供了核苷酸和氨基酸形式的新型腺相关病毒（AAV）分离株及其用途。 分离株对于某些靶组织，如血液干细胞，肝脏，心脏和关节组织都显示向性，并且可用于转导干细胞以将目标组织中感兴趣的基因引入。 帽基因VP1，VP2和VP3的离散修饰部分可以单独使用或在本方法中组合使用。

公开(公告)号：US20240082288A1

公开(公告)日：2024-03-14

申请号：US18450130

申请日：2023-08-15

Applicant: The Trustees of The University of Pennsylvania

Inventor： James M. Wilson ,  Christie L. Bell ,  Luc H. Vandenberghe

IPC: A61K31/7088 ,  A61K31/685 ,  A61K38/47 ,  C12N15/86

CPC classification number: A61K31/7088 ,  A61K31/685 ,  A61K38/47 ,  C12N15/86 ,  C12N2750/14043 ,  C12N2750/14045 ,  C12N2810/6027

Abstract: A method of altering the targeting and/or cellular uptake efficiency of an adeno-associated virus (AAV) viral vector having a capsid containing an AAV9 cell surface binding domain is described. The method involves modifying a clade F cell surface receptor which comprises a glycan having a terminal sialic acid residue and a penultimate β-galactose residue. The modification may involve retargeting the vector by temporarily functionally ablate AAV9 binding in a subset of cells, thereby redirecting the vector to another subset of cells. Alternatively, the modification may involve increasing cellular update efficiency by treating the cells with a neuraminidase to expose cell surface β-galactose. Also provided are compositions containing the AAV9 vector and a neuraminidase. Also provided is a method for purifying AAV9 using β-galactose linked to solid support. Also provided are mutant vectors which have been modified to alter their targeting specificity, including mutant AAV9 in which the galactose binding domain is mutated and AAV in which an AAV9 galactose binding domain is engineered.

公开(公告)号：US11702672B2

公开(公告)日：2023-07-18

申请号：US16076219

申请日：2017-02-08

Applicant: University of Iowa Research Foundation ,  University of Kansas

Inventor： Ziying Yan ,  John F. Engelhardt ,  Jianming Qiu

IPC: C12N15/63 ,  C12N15/85 ,  C07H21/04 ,  C12N15/86 ,  C07K14/015

CPC classification number: C12N15/86 ,  C07K14/015 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2810/6027

Abstract: A method of preparing a chimeric virus comprising bocavirus capsid protein (VP) and a recombinant adeno-associated (AAV) viral genome, and isolated mutant bocavirus genomes, are provided.

公开(公告)号：US20180230490A1

公开(公告)日：2018-08-16

申请号：US15858303

申请日：2017-12-29

Applicant: Genzyme Corporation

Inventor： Catherine O'RIORDAN ,  Samuel WADSWORTH

IPC: C12N15/86 ,  A61K38/17 ,  A61K48/00

CPC classification number: C12N15/86 ,  A61K38/1709 ,  A61K48/005 ,  A61K48/0075 ,  C07K2319/00 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/6027 ,  C12N2830/002 ,  C12N2830/15

Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant viral vector containing the transgene to the spinal cord. The viral vector delivers the transgene which expresses the encoded recombinant viral gene product. The viral gene product comprises HIF1-alpha. Also provided are compositions for delivery of a transgene product to a subject's spinal cord.

公开(公告)号：US10011640B2

公开(公告)日：2018-07-03

申请号：US14214011

申请日：2014-03-14

Applicant: University of Florida Research Foundation, Incorporated

Inventor： Arun Srivastava ,  George Vladimirovich Aslanidi ,  Sergei Zolotukhin ,  Mavis Agbandje-McKenna ,  Kim M. Van Vliet ,  Li Zhong ,  Lakshmanan Govindasamy

IPC: C12N15/86 ,  C07K14/005 ,  A61K38/42 ,  C07K14/805

CPC classification number: C07K14/005 ,  A61K38/42 ,  C07K14/805 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  C12N2810/6027

Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.

公开(公告)号：US09834789B2

公开(公告)日：2017-12-05

申请号：US15465387

申请日：2017-03-21

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: A61K48/00 ,  C12N15/86 ,  C07K14/005 ,  C12N7/00

CPC classification number: A61K48/005 ,  A61K48/00 ,  A61K48/0075 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2710/16043 ,  C12N2750/14122 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2810/6027

Abstract: Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.

公开(公告)号：US09828587B2

公开(公告)日：2017-11-28

申请号：US14782876

申请日：2014-04-08

Applicant: University of Iowa Research Foundation ,  University of Kansas

Inventor： Ziying Yan ,  John F. Engelhardt ,  Jianming Qiu

IPC: A61K39/12 ,  A61K39/23 ,  C12N15/00 ,  C07K1/00 ,  C12N7/00 ,  A61K39/00 ,  C12N15/86 ,  C12N15/864 ,  C07K14/435

CPC classification number: C12N7/00 ,  A61K39/00 ,  A61K39/23 ,  A61K2039/5256 ,  C07K14/435 ,  C12N15/86 ,  C12N15/8645 ,  C12N2750/14111 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2750/14144 ,  C12N2750/14145 ,  C12N2750/14322 ,  C12N2750/14333 ,  C12N2810/6027

Abstract: The invention provides an isolated chimeric virus comprising bocavirus capsid protein, e.g., an isolated chimeric virus comprising human bocavirus capsid protein, and a recombinant adeno-associated viral (AAV) genome, an isolated recombinant bocavirus (rBoV) comprising human bocavirus capsid protein and a recombinant Boy genome, and uses therefor, for example, in gene therapy for diseases in a mammal including diseases with aberrant expression of an endogenous gene product.

公开(公告)号：US20170296677A1

公开(公告)日：2017-10-19

申请号：US15330826

申请日：2016-11-07

Applicant: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

Inventor： Benhur Lee ,  Karina Palomares ,  Olivier Pernet

IPC: A61K48/00 ,  C12N15/86 ,  C12N7/00

CPC classification number: A61K48/0008 ,  A61K39/12 ,  A61K48/00 ,  A61K48/0075 ,  C12N7/00 ,  C12N15/86 ,  C12N2740/15021 ,  C12N2740/15032 ,  C12N2740/15041 ,  C12N2740/15043 ,  C12N2740/15071 ,  C12N2760/18222 ,  C12N2760/18232 ,  C12N2760/18234 ,  C12N2760/18241 ,  C12N2760/18245 ,  C12N2760/18271 ,  C12N2810/6027 ,  C12N2810/6072

Abstract: The present invention relates to lentiviral particles which have been pseudotyped with Nipah virus (NiV) fusion (F) and attachment (G) glycoproteins (NiVpp-F/G). Additionally, the present invention relates to truncated NiV-F glyocproteins useful in producing such NiVpp lentiviral particles, as well as to additional variant peptides which enhance activity. Further, the present invention relates to methods of using such lentiviral particles or sequences, for example in the treatment of cancer or CNS disorders.

公开(公告)号：US09783825B2

公开(公告)日：2017-10-10

申请号：US15479010

申请日：2017-04-04

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: A61K48/00 ,  C12N15/86 ,  C07K14/005 ,  C12N7/00

CPC classification number: A61K48/005 ,  A61K48/00 ,  A61K48/0075 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2710/16043 ,  C12N2750/14122 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2810/6027

Abstract: Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.