公开(公告)号：US12023386B2

公开(公告)日：2024-07-02

申请号：US17563633

申请日：2021-12-28

Applicant: The Trustees of the University of Pennsylvania

Inventor： Anna P. Tretiakova ,  Jenny Agnes Sidrane ,  James M. Wilson

IPC: C12N15/63 ,  A61K35/761 ,  A61K48/00 ,  A61P1/16 ,  C12N9/10 ,  C12N15/62

CPC classification number: A61K48/0058 ,  A61K35/761 ,  A61K48/0008 ,  A61P1/16 ,  C12N9/1051 ,  C12N15/62 ,  C12Y204/01017 ,  C12N2750/14143 ,  C12N2810/6027 ,  C12N2830/008

Abstract: The invention provides a polynucleotide sequence (e.g., a gene, e.g., DNA or RNA) encoding UGT1A1 (e.g., expressing human UGT1A1). The invention further provides a vector, such as an adeno-associated virus (AAV) vector (e.g., AAV8) having a vector genome including inverted terminal repeat sequences and a UGT1A1 coding sequence operably linked to one or more expression control sequences (e.g., expression control sequences including a liver-specific promoter). Also provided are compositions containing these AAV vectors and methods of treating Crigler-Nijjar syndrome type I, Crigler-Nijjar syndrome type II, and Gilbert syndrome.

公开(公告)号：US20240002882A1

公开(公告)日：2024-01-04

申请号：US18313661

申请日：2023-05-08

Applicant: University of Iowa Research Foundation ,  University of Kansas

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

IPC: 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：US11771763B2

公开(公告)日：2023-10-03

申请号：US16455077

申请日：2019-06-27

Applicant: EOS Neuroscience, Inc. ,  Wake Forest University Health Sciences

Inventor： Alan Horsager ,  Kenneth Greenberg ,  Benjamin C. Matteo ,  Edward S. Boyden ,  Douglas G. Ririe ,  James C. Eisenach ,  Christian Wentz

IPC: A61N5/06 ,  A61K48/00 ,  A61K49/00 ,  A61K41/00 ,  C07K14/195 ,  C07K14/37 ,  A61K31/7088 ,  C07K14/215

CPC classification number: A61K41/00 ,  A61K31/7088 ,  A61N5/062 ,  C07K14/195 ,  C07K14/215 ,  C07K14/37 ,  C07K2319/60 ,  C12N2750/14143 ,  C12N2810/6027

Abstract: The present invention provides compositions and methods for the selective silencing of neurons in pain pathway by using a combination of inhibitory light-sensitive protein gene transfer and wavelength specific illumination.

公开(公告)号：US20180051299A9

公开(公告)日：2018-02-22

申请号：US14994477

申请日：2016-01-13

Applicant: University of Florida Research Foundation, Inc.

Inventor： Barry John Byrne ,  Darin J. Falk ,  Christina Pacak ,  Lara Robert Deruisseau ,  Cathryn Mah ,  David D. Fuller

IPC: C12N15/86 ,  A61K9/00 ,  C12N7/00

CPC classification number: C12N15/86 ,  A61K9/0019 ,  A61K45/06 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N2710/16644 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14151 ,  C12N2810/6027 ,  C12N2830/008 ,  C12Y302/0102

Abstract: The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

公开(公告)号：US09839696B2

公开(公告)日：2017-12-12

申请号：US15454754

申请日：2017-03-09

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: A61K48/00 ,  C12N15/86 ,  C12N7/00 ,  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：US09775918B2

公开(公告)日：2017-10-03

申请号：US14847528

申请日：2015-09-08

Applicant: University of Florida Research Foundation, Inc.

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

IPC: A61K48/00 ,  C12N15/86 ,  C12N15/864 ,  A61K35/76 ,  C12N7/00

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 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.

公开(公告)号：US20170274024A1

公开(公告)日：2017-09-28

申请号：US15488966

申请日：2017-04-17

Applicant: The University of North Carolina at Chapel Hill

Inventor： Thomas McCown ,  Steven Gray

IPC: A61K35/76 ,  A61K9/00 ,  C07K14/005 ,  C12N15/86 ,  C12N7/00 ,  A61K39/23 ,  C07K14/015 ,  C12N15/864

CPC classification number: A61K35/76 ,  A61K9/0085 ,  A61K39/23 ,  C07K14/005 ,  C07K14/015 ,  C12N7/00 ,  C12N15/86 ,  C12N15/8645 ,  C12N2750/14122 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/6027

Abstract: The invention relates to chimeric AAV capsids targeted to oligodendrocytes, virus vectors comprising the same, and methods of using the vectors to target oligodendrocytes.

公开(公告)号：US20170211092A1

公开(公告)日：2017-07-27

申请号：US15454754

申请日：2017-03-09

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha Wong

IPC: C12N15/86 ,  C07K14/005 ,  A61K48/00 ,  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：US20170073703A1

公开(公告)日：2017-03-16

申请号：US15337499

申请日：2016-10-28

Applicant: CITY OF HOPE

Inventor： Saswati Chatterjee ,  Laura Jane Smith ,  Kamehameha K. Wong, JR.

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

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

Abstract translation: 提供了核苷酸和氨基酸形式的新型腺相关病毒（AAV）载体及其用途。 分离物显示某些靶组织如血液干细胞，肝脏，心脏和关节组织的特异性向性，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。 某些载体能够交叉紧密控制的生物结，例如血脑屏障，其开启额外的新用途和用于载体的靶器官，提供基因治疗和药物递送的其它方法。

公开(公告)号：US09585971B2

公开(公告)日：2017-03-07

申请号：US14485024

申请日：2014-09-12

Applicant: California Institute of Technology

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

IPC: C07K7/06 ,  C12N15/85 ,  C12N7/00 ,  A61K38/50 ,  A61K38/47 ,  A61K38/00 ,  A61K48/00 ,  A61K38/17 ,  A61K38/20 ,  A61K38/48 ,  A61K39/395 ,  C12N15/86

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.

Abstract translation: 本文提供了选择性筛选的方法。 此外，还提供了各种靶向蛋白和序列，以及它们的使用方法。