公开(公告)号：US20210187006A1

公开(公告)日：2021-06-24

申请号：US17191864

申请日：2021-03-04

Applicant: Regents of the University of Minnesota

Inventor： Mark Osborn ,  Jakub Tolar ,  Bruce Robert Blazar ,  Daniel Voytas

IPC: A61K31/713 ,  A61K31/7088 ,  C12N9/16 ,  C12N9/22

Abstract: The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

公开(公告)号：US20180142262A1

公开(公告)日：2018-05-24

申请号：US15820653

申请日：2017-11-22

Applicant: REGENTS OF THE UNIVERSITY OF MINNESOTA

Inventor： Beau R. Webber ,  Mark J. Osborn ,  Jakub Tolar ,  Bruce R. Blazar

IPC: C12N15/90 ,  C12N5/077 ,  C12N5/074 ,  C12N5/071 ,  C07K14/78

CPC classification number: C12N15/907 ,  C07K14/78 ,  C07K2319/81 ,  C12N5/0629 ,  C12N5/0647 ,  C12N5/0652 ,  C12N5/0656 ,  C12N5/0696 ,  C12N15/102 ,  C12N2500/38 ,  C12N2500/90 ,  C12N2501/155 ,  C12N2501/727 ,  C12N2502/28 ,  C12N2506/1307 ,  C12N2506/45 ,  C12N2510/00 ,  C12N2533/54 ,  C12N2760/18843

Abstract: Methods of gene correction, methods of generating induced pluripotent stem cells (iPSCs), and methods of deriving multi-lineage cell types with therapeutic value. In some embodiments, the gene correction affects the expression and/or function of the functional type VII collagen protein (C7).

公开(公告)号：US20170175143A1

公开(公告)日：2017-06-22

申请号：US15311685

申请日：2015-05-20

Applicant: REGENTS OF THE UNIVERSITY OF MINNESOTA

Inventor： Jakub Tolar ,  Bruce Robert Blazar ,  Daniel Francis Voytas ,  Mark John Osborn

IPC: C12N15/90 ,  C12N5/0789 ,  A61K35/28 ,  C12N9/22

CPC classification number: C12N15/907 ,  A61K35/28 ,  A61K2035/124 ,  C12N5/0647 ,  C12N9/22 ,  C12N15/102

Abstract: This disclosure describes methods, polynucleotides, cells, compositions, and treatment methods that involve channeling a genomic nucleotide sequence. Generally, the method includes introducing a donor polynucleotide and a nucleotide that encodes an enzyme that cuts at least one strand of DNA into a cell that has a genomic sequence in need of editing, allowing the enzyme to cut at least one strand of the genomic sequence, and allowing the donor sequence to replace the genomic sequence in need of editing.

公开(公告)号：US20200248139A1

公开(公告)日：2020-08-06

申请号：US16637450

申请日：2018-08-08

Applicant: REGENTS OF THE UNIVERSITY OF MINNESOTA

Inventor： Timothy D. O'Brien ,  Beth A. Lindborg ,  Amanda Vegoe ,  Jakub Tolar

IPC: C12N5/079 ,  A61K35/30

Abstract: This disclosure describes methods for organoid generation including, for example, for generation of a mid-brain organoid including an A9 neuron. Specifically, the methods comprising: introducing an input cell into a cell culture medium comprising hyaluronic acid, wherein the input cell comprises an embryonic stem cell, an induced pluripotent stem cell, or a neural progenitor cell; transferring the input cell to a cell culture device; and culturing the cell in the cell culture device for at least 7 days. This disclosure further describes methods for using the organoids.

公开(公告)号：US09393257B2

公开(公告)日：2016-07-19

申请号：US14193037

申请日：2014-02-28

Applicant: Regents of the University of Minnesota

Inventor： Mark J. Osborn ,  Jakub Tolar ,  Bruce Blazar ,  Daniel F. Voytas

IPC: A61K31/7088 ,  C12N9/16

CPC classification number: A61K31/713 ,  A61K31/7088 ,  C12N9/16

Abstract: The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

Abstract translation: 本发明涉及在人类基因组和人类细胞的上下文中的转录激活子样效应子核酸酶（TALEN）介导的DNA编辑疾病引起的突变，以治疗患有遗传障碍的患者。

公开(公告)号：US12270050B2

公开(公告)日：2025-04-08

申请号：US16060651

申请日：2016-12-08

Applicant: Regents of the University of Minnesota

Inventor： Bruce R. Blazar ,  Dharmeshkumar Patel ,  Beau R. Webber ,  Jakub Tolar

IPC: A61K35/545 ,  A61K35/17 ,  A61K39/00 ,  C12N5/074 ,  C12N5/0783

Abstract: Induced pluripotent stem cells (iPSCs) derived from a T cell of a T cell subset. T cells derived from iPSCs derived from a T cell. Methods of deriving iPSCs from a T cell. Methods of deriving T cells from iPSCs including deriving a T cell of a T cell subset from an iPSC. Methods of engineering chimeric antigen receptor (CAR)-expressing or T cell receptor (TCR)-expressing iPSC. Methods of administering T cells derived using the methods disclosed. Induced pluripotent stem cell lines derived from T cells, methods of deriving induced pluripotent stem cell lines, and methods of deriving T cells from induced pluripotent stem cell lines.

公开(公告)号：US20160367588A1

公开(公告)日：2016-12-22

申请号：US15182773

申请日：2016-06-15

Applicant: Regents of the University of Minnesota

Inventor： Mark Osborn ,  Jakub Tolar ,  Bruce Robert Blazar ,  Daniel Voytas

IPC: A61K31/713

CPC classification number: A61K31/713 ,  A61K31/7088 ,  C12N9/16

Abstract: The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

Abstract translation: 本发明涉及在人类基因组和人类细胞的上下文中的转录激活子样效应子核酸酶（TALEN）介导的DNA编辑疾病致突变，以治疗患有遗传障碍的患者。

公开(公告)号：US20230374451A1

公开(公告)日：2023-11-23

申请号：US18028180

申请日：2021-09-23

Applicant: REGENTS OF THE UNIVERSITY OF MINNESOTA

Inventor： Elizabeth Louise Thompson ,  Jakub Tolar

IPC: C12N5/071 ,  C07K14/025 ,  C07K14/435 ,  C12N9/12 ,  C12N15/86

CPC classification number: C12N5/0629 ,  C07K14/025 ,  C07K14/435 ,  C12N9/1276 ,  C12N15/86 ,  C12Y207/07049 ,  C12N2513/00 ,  C12N2500/14 ,  C12N2501/105 ,  C12N2501/39 ,  C12N2501/11 ,  C12N2500/24 ,  C12N2740/15022

Abstract: Provided herein are recombinant lentiviral vectors and methods of using the same to produce genetically modified keratinocytes that can be maintained in culture for over 100 population doubling without loss of morphological features or differentiation capacity. Immortalized keratinocytes and immortalized cell lines obtained by the methods of this disclosure are useful for studying the role of pathogenic mutations in skin disease phenotype, for screening for potential therapeutic agents, and for producing race-, sex, and age-specific epidermis for research and clinical applications.

公开(公告)号：US10973844B2

公开(公告)日：2021-04-13

申请号：US16176073

申请日：2018-10-31

Applicant: Regents of the University of Minnesota

Inventor： Mark Osborn ,  Jakub Tolar ,  Bruce Robert Blazar ,  Daniel Voytas

IPC: A61K31/713 ,  A61K31/7088 ,  C12N9/16 ,  C12N9/22

Abstract: The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

公开(公告)号：US20200165574A1

公开(公告)日：2020-05-28

申请号：US16612900

申请日：2018-05-11

Applicant: Jakub Tolar ,  Kirk Robert Twaroski ,  Emily Faith Ward ,  James Dutton ,  REGENTS OF THE UNIVERSITY OF MINNESOTA

Inventor： Jakub Tolar ,  Kirk Robert Twaroski ,  Emily Faith Ward ,  James Dutton

IPC: C12N5/074

Abstract: Methods of preparing naive human pluripotent stem cells are described. The methods include the use of xeno-free media and do not include the use of feeder cells.