公开(公告)号：US11939355B2

公开(公告)日：2024-03-26

申请号：US17209336

申请日：2021-03-23

Applicant: Howard Hughes Medical Institute ,  The Regents of the University of California

Inventor： Joshua Dudman ,  Adam Hantman ,  Bum-Yeol Hwang ,  Alla Karpova ,  Loren Looger ,  Kimberly Ritola ,  David Schaffer ,  Dougal Gowanlock Robinson Tervo ,  Sarada Viswanathan

IPC: C12N7/00 ,  C07K14/005 ,  C07K14/015 ,  C12N15/86 ,  C40B30/06 ,  C40B40/08 ,  A61K48/00

CPC classification number: C07K14/015 ,  C07K14/005 ,  C12N15/86 ,  C40B30/06 ,  C40B40/08 ,  A61K48/00 ,  C12N2750/14122 ,  C12N2750/14145 ,  C12N2810/6027

Abstract: The present disclosure provides AAV variants that exhibit a preference for retrograde movement in neurons and methods of using such variants.

公开(公告)号：US11833226B2

公开(公告)日：2023-12-05

申请号：US16787980

申请日：2020-02-11

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: C12N15/85 ,  A61K48/00 ,  C12N15/86 ,  C07K14/005 ,  C12N7/00

CPC classification number: A61K48/005 ,  A61K48/0075 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00 ,  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：US20230285591A1

公开(公告)日：2023-09-14

申请号：US18151174

申请日：2023-01-06

Applicant: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

Inventor： Benhur LEE ,  Karina PALOMARES ,  Olivier PERNET

IPC: A61K48/00 ,  A61K39/12 ,  C12N7/00 ,  C12N15/86

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 realtes 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 treatetment of cancer or CNS disorders.

公开(公告)号：US20180185405A1

公开(公告)日：2018-07-05

申请号：US15862999

申请日：2018-01-05

Applicant: The Trustees of The University of Pennsylvania

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

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

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：US20180163227A1

公开(公告)日：2018-06-14

申请号：US15797868

申请日：2017-10-30

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: C12N15/86 ,  A61K48/00 ,  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：US09957303B2

公开(公告)日：2018-05-01

申请号：US15422237

申请日：2017-02-01

Applicant: California Institute of Technology

Inventor： Benjamin E. Deverman ,  Paul H. Patterson ,  Viviana Gradinaru

IPC: C07K14/005 ,  C07K7/06 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00

CPC classification number: C07K14/005 ,  A61K38/00 ,  A61K38/1709 ,  A61K38/2093 ,  A61K38/47 ,  A61K38/4813 ,  A61K38/50 ,  A61K39/3955 ,  A61K48/005 ,  A61K48/0058 ,  C07K7/06 ,  C07K2319/33 ,  C12N7/00 ,  C12N15/1068 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/6027 ,  C12Y304/14009 ,  C12Y305/01015

Abstract: Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.

公开(公告)号：US09920097B2

公开(公告)日：2018-03-20

申请号：US13899481

申请日：2013-05-21

Applicant: University of Florida Research Foundation, Inc.

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

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

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 capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more symptoms of disease or abnormal conditions via in situ and/or ex vivo mammalian gene therapy methods. 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：US09890394B2

公开(公告)日：2018-02-13

申请号：US12417910

申请日：2009-04-03

Applicant: Catherine O'Riordan ,  Samuel Wadsworth

Inventor： Catherine O'Riordan ,  Samuel Wadsworth

IPC: A61K31/70 ,  C12N15/00 ,  A61K39/00 ,  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：US20170211093A1

公开(公告)日：2017-07-27

申请号：US15465378

申请日：2017-03-21

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: C12N15/86 ,  C07K14/005

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：US20170204144A1

公开(公告)日：2017-07-20

申请号：US15422237

申请日：2017-02-01

Applicant: California Institute of Technology

Inventor： Benjamin E. Deverman ,  Paul H. Patterson ,  Viviana Gradinaru

IPC: C07K14/005 ,  A61K48/00 ,  C12N15/86 ,  C07K7/06 ,  C12N7/00

CPC classification number: C07K14/005 ,  A61K38/00 ,  A61K38/1709 ,  A61K38/2093 ,  A61K38/47 ,  A61K38/4813 ,  A61K38/50 ,  A61K39/3955 ,  A61K48/005 ,  A61K48/0058 ,  C07K7/06 ,  C07K2319/33 ,  C12N7/00 ,  C12N15/1068 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/6027 ,  C12Y304/14009 ,  C12Y305/01015

Abstract: Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.