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公开(公告)号:US20190144845A9
公开(公告)日:2019-05-16
申请号:US16029273
申请日:2018-07-06
Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventor: Hao Yin , Wen Xue , Daniel G. Anderson , Joseph R. Dorkin , Tyler E. Jacks
Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
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2.
公开(公告)号:US09567642B2
公开(公告)日:2017-02-14
申请号:US14376482
申请日:2013-02-01
Applicant: Massachusetts Institute of Technology
Inventor: David Feldser , Tyler E. Jacks , Leah Marie Schmidt
IPC: A61K39/395 , C12Q1/68 , G01N33/574 , A61K45/06 , C07K16/28
CPC classification number: C12Q1/6886 , A61K39/39558 , A61K45/06 , C07K16/2827 , C12Q2600/106 , G01N33/57484 , G01N2800/52
Abstract: The invention relates to methods and related products for treatment and determining modes of treatment for cancer or prognosis of cancer. Preferably the methods are related to the induction of CD80 in p53 responsive cancer cells.
Abstract translation: 本发明涉及用于治疗和确定癌症治疗方式或癌症预后的方法和相关产品。 优选地,所述方法与p53反应性癌细胞中CD80的诱导有关。
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公开(公告)号:US20190002869A1
公开(公告)日:2019-01-03
申请号:US16029273
申请日:2018-07-06
Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventor: Hao Yin , Wen Xue , Daniel G. Anderson , Joseph R. Dorkin , Tyler E. Jacks
Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
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公开(公告)号:US10047355B2
公开(公告)日:2018-08-14
申请号:US15374227
申请日:2016-12-09
Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventor: Hao Yin , Wen Xue , Daniel G. Anderson , Joseph R. Dorkin , Tyler E. Jacks
Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
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公开(公告)号:US20210338789A1
公开(公告)日:2021-11-04
申请号:US17198037
申请日:2021-03-10
Inventor: Omar F. Khan , Jasdave S. Chahal , Daniel G. Anderson , Hidde Ploegh , Robert S. Langer , Tyler E. Jacks , David A. Canner
IPC: A61K39/00 , A61K31/713 , A61K39/12 , C12N15/87 , A61K48/00 , A61K39/002 , A61K9/00 , C12N15/88 , A61K39/145 , A61K9/51
Abstract: Compositions and methods for modified dendrimer nanoparticle (“MDNP”) delivery of therapeutic, prophylactic and/or diagnostic agent such as large repRNA molecules to the cells of a subject have been developed. MDNPs efficiently drive proliferation of antigen-specific T cells against intracellular antigen, and potentiate antigen-specific antibody responses. MDNPs can be multiplexed to deliver two or more different repRNAs to modify expression kinetics of encoded antigens and to simultaneous deliver repRNAs and mRNAs including the same UTR elements that promote expression of encoded antigens.
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公开(公告)号:US10941395B2
公开(公告)日:2021-03-09
申请号:US16029273
申请日:2018-07-06
Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventor: Hao Yin , Wen Xue , Daniel G. Anderson , Joseph R. Dorkin , Tyler E. Jacks
Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
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公开(公告)号:US20170015994A1
公开(公告)日:2017-01-19
申请号:US15121292
申请日:2015-02-24
Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventor: Daniel G. Anderson , Hao Yin , Roman L. Bogorad , Tyler E. Jacks , Wen Xue
IPC: C12N15/10 , A61K31/713 , A61K9/00
CPC classification number: C12N15/1024 , A61K9/0019 , A61K31/713 , C12N15/102 , C12N15/63
Abstract: Methods of transfecting cells in vivo, by administering an injectable pharmaceutical composition including a genome editing composition and a pharmaceutically acceptable carrier to a subject by hydrodynamic injection into a vessel of the subject are disclosed. Typically, the pharmaceutical composition is administered in a volume and at rate of injection suitable to transfect target eukaryotic cells in the subject with an effective amount of the genome editing composition to alter the genome of the target cells. In preferred embodiments the subject is a mammal, such as rodent, or a primate such as a human. The methods can be used to treat one or more symptoms of a genetic disease or condition.
Abstract translation: 公开了通过向受试者施用包含基因组编辑组合物和药学上可接受的载体的可注射药物组合物在体内转染细胞的方法,通过流体动力学注射到受试者的血管中。 通常,药物组合物以体积和注射速率施用,适合于用有效量的基因组编辑组合物转染受试者中的靶真核细胞以改变靶细胞的基因组。 在优选的实施方案中,受试者是哺乳动物,例如啮齿动物,或灵长类动物例如人。 该方法可用于治疗遗传性疾病或病症的一种或多种症状。
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公开(公告)号:US12163124B2
公开(公告)日:2024-12-10
申请号:US17195625
申请日:2021-03-08
Applicant: Massachusetts Institute of Technology
Inventor: Hao Yin , Wen Xue , Daniel G. Anderson , Joseph R. Dorkin , Tyler E. Jacks
Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
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公开(公告)号:US20210324370A1
公开(公告)日:2021-10-21
申请号:US17195625
申请日:2021-03-08
Applicant: Massachusetts Institute of Technology
Inventor: Hao Yin , Wen Xue , Daniel G. Anderson , Joseph R. Dorkin , Tyler E. Jacks
Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for deliverying such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
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公开(公告)号:US20200155660A1
公开(公告)日:2020-05-21
申请号:US16692624
申请日:2019-11-22
Inventor: Omar F. Khan , Jasdave S. Chahal , Daniel G. Anderson , Hidde Ploegh , Robert S. Langer , Tyler E. Jacks , David A. Canner
IPC: A61K39/00 , A61K31/713 , A61K39/12 , C12N15/87 , A61K48/00 , A61K39/002 , A61K9/00 , C12N15/88 , A61K39/145 , A61K9/51
Abstract: Compositions and methods for modified dendrimer nanoparticle (“MDNP”) delivery of therapeutic, prophylactic and/or diagnostic agent such as large repRNA molecules to the cells of a subject have been developed. MDNPs efficiently drive proliferation of antigen-specific T cells against intracellular antigen, and potentiate antigen-specific antibody responses. MDNPs can be multiplexed to deliver two or more different repRNAs to modify expression kinetics of encoded antigens and to simultaneous deliver repRNAs and mRNAs including the same UTR elements that promote expression of encoded antigens.
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