公开(公告)号：US20180030438A1

公开(公告)日：2018-02-01

申请号：US15550951

申请日：2016-02-23

Applicant: CRISPR Therapeutics AG ,  Georgia Tech Research Corporation ,  William Marsh Rice University

Inventor： Matthew Hebden Porteus ,  Thomas James Cradick ,  Gang Bao ,  Ciaran M. Lee

IPC: C12N15/113 ,  C12N15/63

CPC classification number: C12N15/113 ,  A61K35/28 ,  A61K35/545 ,  A61K38/42 ,  A61K48/00 ,  C12N5/0647 ,  C12N5/0696 ,  C12N15/102 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2501/73 ,  C12N2510/00

Abstract: The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

公开(公告)号：US20170053062A1

公开(公告)日：2017-02-23

申请号：US15114799

申请日：2015-01-27

Applicant: GEORGIA TECH RESEARCH CORPORATION

Inventor： Thomas James Cradick ,  Gang Bao ,  Peng Qiu

IPC: G06F19/22 ,  G06F17/30 ,  G06N7/00

CPC classification number: G16B30/00 ,  G06F16/2228 ,  G06F16/2468 ,  G06N7/005

Abstract: Methods and systems for searching genomes for potential CRISPR off-target sites are provided. In preferred embodiments, the methods include identifying possible on- and off-target cleavage sites and/or ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the gRNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome. These methods allow for the selection of better target sites and/or experimental confirmation of off-target sites and are an improvement over partial search mechanisms that fail to locate every possible target site.

Abstract translation: 提供了用于搜索潜在CRISPR离靶位点的基因组的方法和系统。 在优选的实施方案中，所述方法包括基于在gRNA指导序列中的错配，插入和/或缺失的数量和位置，相对于 基因组DNA序列在基因组中的推定靶位点。 这些方法允许选择更好的目标站点和/或非目标站点的实验确认，并且是对部分搜索机制的改进，这些机制无法定位每个可能的目标站点。

公开(公告)号：US11315659B2

公开(公告)日：2022-04-26

申请号：US16594905

申请日：2019-10-07

Applicant: Georgia Tech Research Corporation

Inventor： Thomas James Cradick ,  Gang Bao ,  Peng Qiu

IPC: G16B30/00 ,  G06F16/22 ,  G06F16/2458 ,  G06N7/00

Abstract: Methods and systems for searching genomes for potential nucleotide-guided nuclease off-target sites are provided. Also provided are methods of searching genomes for potential off-target deadCas9 binding sites. In some embodiments, the methods include ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the DNA, RNA, or DNA/RNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome, allowing the selection of better target sites and/or experimental confirmation of off-target sites.

公开(公告)号：US10354746B2

公开(公告)日：2019-07-16

申请号：US15114799

申请日：2015-01-27

Applicant: Georgia Tech Research Corporation

Inventor： Thomas James Cradick ,  Gang Bao ,  Peng Qiu

IPC: G06F17/30 ,  G16B30/00 ,  G06F16/22 ,  G06F16/2458 ,  G06F19/22 ,  G06N7/00

Abstract: Methods and systems for searching genomes for potential CRISPR off-target sites are provided. In preferred embodiments, the methods include identifying possible on- and off-target cleavage sites and/or ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the gRNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome. These methods allow for the selection of better target sites and/or experimental confirmation of off-target sites and are an improvement over partial search mechanisms that fail to locate every possible target site.

公开(公告)号：US20200058373A1

公开(公告)日：2020-02-20

申请号：US16594905

申请日：2019-10-07

Applicant: Georgia Tech Research Corporation

Inventor： Thomas James Cradick ,  Gang Bao ,  Peng Qiu

IPC: G16B30/00 ,  G06N7/00 ,  G06F16/2458 ,  G06F16/22

Abstract: Methods and systems for searching genomes for potential nucleotide-guided nuclease off-target sites are provided. Also provided are methods of searching genomes for potential off-target deadCas9 binding sites. In some embodiments, the methods include ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the DNA, RNA, or DNA/RNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome, allowing the selection of better target sites and/or experimental confirmation of off-target sites.

公开(公告)号：US20190295689A1

公开(公告)日：2019-09-26

申请号：US16410395

申请日：2019-05-13

Applicant: Georgia Tech Research Corporation

Inventor： Thomas James Cradick ,  Gang Bao ,  Peng Qiu

IPC: G16B30/00 ,  G06F16/2458 ,  G06N7/00 ,  G06F16/22

Abstract: Methods and systems for searching genomes for potential CRISPR off-target sites are provided. In preferred embodiments, the methods include identifying possible on- and off-target cleavage sites and /or ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the gRNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome. These methods allow for the selection of better target sites and/or experimental confirmation of off-target sites and are an improvement over partial search mechanisms that fail to locate every possible target site.