-
公开(公告)号：US20240360452A1
公开(公告)日：2024-10-31
申请号：US18771603
申请日：2024-07-12
IPC分类号： C12N15/113 , A61K31/56 , A61K31/57 , A61K31/573 , A61K31/58 , A61K31/7088 , A61K38/17 , A61K45/06 , A61K48/00
CPC分类号： C12N15/113 , A61K31/56 , A61K31/57 , A61K31/573 , A61K31/58 , A61K31/7088 , A61K38/1719 , A61K45/06 , A61K48/00 , C12N2310/11 , C12N2310/111 , C12N2310/31 , C12N2310/313 , C12N2310/314 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3233 , C12N2310/346 , C12N2320/31 , C12N2320/33
摘要： The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.
-
公开(公告)号：US20240352461A1
公开(公告)日：2024-10-24
申请号：US18765286
申请日：2024-07-07
申请人： YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD. , SpliSense Ltd.
IPC分类号： C12N15/113 , A61K31/7088 , A61K45/06 , A61P11/00
CPC分类号： C12N15/113 , A61K31/7088 , A61K45/06 , A61P11/00 , C12N2310/11 , C12N2320/31
摘要： The present invention provides oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, including compositions comprising the oligonucleotides, and uses thereof, such as for suppressing the inclusion of a cryptic exon between exon 22 and 23 as a result of the mutation 3849+10 Kb C-to-T, optionally in combination with additional CF therapeutics.
-
公开(公告)号：US20240350581A1
公开(公告)日：2024-10-24
申请号：US18288813
申请日：2022-04-29
IPC分类号： A61K38/17 , A61P25/28 , C12N15/113
CPC分类号： A61K38/1709 , A61P25/28 , C12N15/113 , C12N2310/14 , C12N2320/31
摘要： A method for reducing, inhibiting, preventing or treating cytotoxicity in a cell, the method comprising expressing or overexpressing in the cell a Rho guanine nucleotide exchange factor (RGNEF), or a RGNEF analog or agonist, or a leucine rich domain of RGNEF, whereby the cytotoxicity in the cell is reduced, inhibited, prevented or treated. Also a method of shifting the survival curve of a subject and thereby increasing life expectancy of the subject by administering to the subject a physiologically effective amount of a peptide comprising A Rho Guanine Nucleotide Exchange Factor (RGNEF) protein, or a RGNEF analog or agonist, or a leucine rich domain of RGNEF, or expressing or overexpressing RGNEF or a RGNEF analog or agonist or a leucine rich domain of RGNEF in the subject.
-
公开(公告)号：US12104157B2
公开(公告)日：2024-10-01
申请号：US18155163
申请日：2023-01-17
申请人： miRecule, Inc.
发明人： Robert Place , Anthony Saleh , Tishan Williams
IPC分类号： C07H21/04 , A61K31/7088 , A61K47/54 , C12N15/113
CPC分类号： C12N15/113 , A61K31/7088 , A61K47/549 , C12N2310/141 , C12N2310/316 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/351 , C12N2310/3521 , C12N2310/531 , C12N2320/31 , C12N2320/32 , C12N2320/35
摘要： Disclosed herein are engineered oligonucleotides for selective inhibition of polypeptide expression and activity. Also disclosed herein are methods of selectively inhibiting polypeptide expression and activity contacting an engineered oligonucleotide with a polynucleotide encoding the polypeptide.
-
公开(公告)号：US20240301409A1
公开(公告)日：2024-09-12
申请号：US18275900
申请日：2022-01-28
发明人： Longcheng Li , Moorim Kang
IPC分类号： C12N15/113 , A61K31/407 , A61K31/704 , A61K31/713 , A61P35/00
CPC分类号： C12N15/113 , A61K31/407 , A61K31/704 , A61K31/713 , A61P35/00 , C12N2310/312 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2320/31
摘要： Provided is a small activating RNA used for promoting an expression level of a target gene. The small activating RNA is composed of a sense oligonucleotide chain and an antisense oligonucleotide chain. A nucleotide of the sense or antisense oligonucleotide chain has 2′-fluoro, 2′-methoxy, and a phosphorothioate modification to the nucleotide backbone.
-
公开(公告)号：US12077760B2
公开(公告)日：2024-09-03
申请号：US17413550
申请日：2019-12-16
发明人： Gabriel Lopez-Berestein , Paola Amero , Cristian Rodriguez-Aguayo , Rahul Mitra , Anil K. Sood , Vittorio De Franciscis , David Volk , Lokesh Ganesh L. Rao
IPC分类号： C12N15/115 , A61K31/337 , A61K33/243 , A61P35/00
CPC分类号： C12N15/115 , A61K31/337 , A61K33/243 , A61P35/00 , C12N2310/16 , C12N2310/313 , C12N2310/322 , C12N2310/351 , C12N2310/531 , C12N2320/31 , C12N2320/32
摘要： Provided herein are DNA aptamers targeting AXL receptor kinase. The DNA aptamers may comprise a thiophosphate backbone and be chemically modified. Further provided herein are methods of use thereof for the treatment of a disease or disorder, such as cancer.
-
公开(公告)号：US12071622B2
公开(公告)日：2024-08-27
申请号：US18149881
申请日：2023-01-04
发明人： Amriti R. Lulla , Wafik S. El-Deiry
IPC分类号： C12N15/113 , A61K31/7105 , A61K39/395 , C07K16/28 , C12Q1/6886
CPC分类号： C12N15/113 , A61K31/7105 , A61K39/39533 , C07K16/2875 , C12Q1/6886 , C07K2317/76 , C12N2310/141 , C12N2320/31 , C12Q2600/106 , C12Q2600/158 , C12Q2600/178
摘要： The present disclosure provides compositions comprising miR-3132 and one or more pharmaceutical agents that upregulate TNF-related apoptosis-inducing ligand (TRAIL) or activate TRAIL signaling pathway, and methods for treating a cancer comprising administering miR-3132, or a composition comprising miR-3132 and one or more pharmaceutical agents that upregulate TRAIL or activate TRAIL signaling pathway, to a subject.
-
8.发明公开公开(公告)号：US20240279352A1
公开(公告)日：2024-08-22
申请号：US18644720
申请日：2024-04-24
IPC分类号： C07K16/28 , A61K39/00 , A61P35/00 , C12N5/0786 , C12N15/113
CPC分类号： C07K16/2896 , A61P35/00 , C07K16/2803 , C07K16/2827 , C07K16/2878 , C12N5/0645 , C12N15/1138 , A61K2039/505 , A61K2039/507 , C07K2317/76 , C12N2310/14 , C12N2320/31
摘要： The present invention concerns the use of an anti-SIRPa compound able to inhibit the polarization of anti-inflammatory M2-type macrophages and/or favors pro-inflammatory M1-type macrophages. In a preferred embodiment, such compound is used to treat cancer. Interestingly, this invention allows to treat cancer through an indirect pathway involving the immune system.
-
公开(公告)号：US20240240180A1
公开(公告)日：2024-07-18
申请号：US18426444
申请日：2024-01-30
IPC分类号： C12N15/113 , A61K31/713 , A61K35/12 , A61K38/57 , A61K48/00 , C07K14/81 , C12N7/00 , C12N15/67 , C12N15/86 , C12N15/11
CPC分类号： C12N15/113 , A61K31/713 , A61K35/12 , A61K38/57 , A61K48/005 , A61K48/0058 , C07K14/8125 , C12N7/00 , C12N15/67 , C12N15/86 , C12N15/111 , C12N2310/141 , C12N2310/3519 , C12N2320/31 , C12N2750/14121 , C12N2750/14132 , C12N2750/14143
摘要： The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
-
公开(公告)号：US20240229044A9
公开(公告)日：2024-07-11
申请号：US18534884
申请日：2023-12-11
IPC分类号： C12N15/113 , A61K38/16 , A61K38/17 , A61K38/45 , A61K38/48 , A61K38/51 , A61P13/06 , C12N15/86
CPC分类号： C12N15/1138 , A61K38/164 , A61K38/1793 , A61K38/45 , A61K38/4893 , A61K38/51 , A61P13/06 , C12N15/86 , C12Y304/24069 , C12Y401/01015 , C12N2310/11 , C12N2320/31 , C12N2320/32 , C12N2710/16643 , C12N2830/008 , C12N2830/40
摘要： The present invention provides a method and a pharmaceutical composition for the treatment of the NDO comprising the viral expression vector carrying a transcription cassette that harbors transgene(s) inhibiting/silencing neurotransmission or synaptic transmission of afferent neurons.
-
-
-
-
-
-
-
-
-
搜索结果
1415 条记录 (耗时 0.028 秒)
