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公开(公告)号:US12234475B2
公开(公告)日:2025-02-25
申请号:US18049266
申请日:2022-10-24
Applicant: GenVivo, Inc.
Inventor: Brile Chung , Cecilia Roh , Robert G. Johnson, Jr.
IPC: C12N15/86 , A61K31/522 , A61P35/00 , C07H21/04
Abstract: Described herein are compositions comprising a recombinant retroviral vector for delivering a therapeutic comprising a nucleic acid construct comprising a polynucleotide sequence encoding an interleukin or a subunit thereof. Also described herein are methods of using the composition comprising a recombinant retroviral vector described herein for delivering a therapeutic comprising a nucleic acid construct comprising a polynucleotide sequence encoding an interleukin or a subunit thereof to a subject.
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公开(公告)号:US12234455B2
公开(公告)日:2025-02-25
申请号:US17065409
申请日:2020-10-07
Inventor: Fabrizio d′Adda di Fagagna , Francesca Rossiello , Julio Aguado , Corey Jones-Weinert
IPC: C07H21/02 , C07H21/04 , C12N15/113
Abstract: The present invention relates to an oligonucleotide comprising one of the following sequence: (TTAGGG) SEQ ID No. 1, (TAGGGT) SEQ ID No. 2, (AGGGTT) SEQ ID No. 3, (GGGTTA) SEQ ID No. 4, (GGTTAG) SEQ ID No. 5 or (GTTAGG) SEQ ID No. 6 or a complementary sequence thereof or a fragment or a variant or a mixture thereof for use in the treatment and/or prevention of a disease characterized by alternative lengthening of telomeres or a non-cancer condition associated with telomere dysfunction and relative pharmaceutical compositions and to relative pharmaceutical composition and method.
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公开(公告)号:US12233050B2
公开(公告)日:2025-02-25
申请号:US17346036
申请日:2021-06-11
Applicant: City University of Hong Kong
Inventor: Wenjun Xiong , Shuyi Mai
IPC: C12N15/86 , A61K31/21 , A61K31/426 , A61K48/00 , C07H21/04 , C12N15/864
Abstract: A treatment of various eye conditions relating to eye enlargement is disclosed. The conditions can be treated with by inhibiting an upstream protein within the biological pathway or by increasing the expression of a downstream receptor within the same pathway. Inhibition of the upstream protein, sterol regulatory element binding protein (SREBP), has been achieved using small molecule inhibitors or nucleic acid in viral vector and increased expression of the downstream protein, bone morphogenetic protein (BMP), has been achieved by nucleic acid in viral vector. Also disclosed are the small molecule, nucleic acid and the viral vector as well as methods of treating ocular diseases.
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公开(公告)号:US12227744B2
公开(公告)日:2025-02-18
申请号:US17721704
申请日:2022-04-15
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor: Dinah Wen-Yee Sah , Gregory Hinkle , Rene Alvarez , Stuart Milstein , Qingmin Chen
IPC: C07H21/02 , A61K9/00 , A61K31/713 , C07F9/6533 , C07H21/04 , C12N15/11 , C12N15/113
Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.
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公开(公告)号:US20250042932A1
公开(公告)日:2025-02-06
申请号:US18714078
申请日:2022-11-29
Applicant: ATDBio Limited
IPC: C07H21/04 , C07H1/00 , C07H19/073
Abstract: Provided herein is a method of producing a polynucleotide or an analogue or derivative thereof, comprising providing a support-attached nucleoside, nucleotide, polynucleotide or derivative or analogue thereof, which is a compound of formula (I) as defined herein and attached to a solid support at (II), (ii) increasing the chain length of the support-attached nucleoside, nucleotide, polynucleotide or derivative or analogue thereof by introducing one or more additional nucleoside units, nucleotide units and/or analogues or derivatives thereof; and (iii) cleaving the resulting polynucleotide from the solid support by photo-illuminating the compound of formula (I). Also provided are associated compounds, uses, and polynucleotides obtainable by such methods.
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公开(公告)号:US12216121B2
公开(公告)日:2025-02-04
申请号:US16603722
申请日:2018-04-12
Applicant: The University of Chicago
Inventor: Yamuna Krishnan , Krishna Dan , Aneesh T. Veetil , Kasturi Chakraborty
IPC: G01N33/573 , C07H21/04 , G01N21/64
Abstract: Described herein are nucleic acid molecules and complexes useful for spatiotemporally mapping intra-endosmal thiol disulphide exchange. Aspects of the disclosure relate to a composition comprising a first nucleic acid conjugated to a normalization moiety; and a second nucleic acid conjugated to a catalytic substrate; wherein reaction of the substrate with a catalyst produces a detectable product; and wherein the first and second nucleic acids are complementary or substantially complementary. Further aspects relate to a composition comprising: a first nucleic acid conjugated to a normalization moiety and to a catalytic substrate; and a second nucleic acid; wherein reaction of the substrate with a catalyst produces a detectable product; and wherein the first and second nucleic acids are complementary or substantially complementary.
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公开(公告)号:US12215124B2
公开(公告)日:2025-02-04
申请号:US16969879
申请日:2019-02-20
Applicant: SINGULAR GENOMICS SYSTEMS, INC.
Inventor: Ronald Graham , Andrew Spaventa , Eli Glezer
IPC: C07H21/04 , C12Q1/6869
Abstract: Disclosed herein, inter alia, are compounds, compositions, and methods of use thereof in the sequencing a nucleic acid.
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公开(公告)号:US20250025578A1
公开(公告)日:2025-01-23
申请号:US18905388
申请日:2024-10-03
Inventor: Paul Taylor Martin
Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.
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公开(公告)号:US12203073B2
公开(公告)日:2025-01-21
申请号:US17129463
申请日:2020-12-21
Applicant: CASE WESTERN RESERVE UNIVERSITY
Inventor: Nicole F. Steinmetz
Abstract: A nanoparticle includes an icosahedral-shaped plant virus particle; an RNAi construct; and one or more endolysosomal release agents, wherein the RNAi construct is noncovalently loaded within the icosahedral-shaped plant virus particle.
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公开(公告)号:US12203070B2
公开(公告)日:2025-01-21
申请号:US16881919
申请日:2020-05-22
Inventor: Eric Kmiec , Pawel Bialk
IPC: C07H21/04 , A61K31/7105 , C12N9/22 , C12N15/113
Abstract: The disclosure provides a guide RNA (gRNA) comprising a DNA-binding domain and a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease protein-binding domain, wherein the DNA-binding domain is complementary to a target domain from an NRF2 gene. The disclosure also provides nucleic acid sequence encoding the gRNA. The disclosure further provides a method of treating cancer in a subject comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease and a guide RNA that is complementary to a target domain from an NRF2 gene in the subject. Methods of treating cancer comprising administering a pharmaceutical composition comprising: a DNA sequence encoding a guide RNA that is complementary to a target domain from an NRF2 gene in the subject; and a nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, are also provided.
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