CAS: 1227633-49-9; 4-(2-((2-(Benzo[b]Thiophen-3-yl)-9-Isopropyl-9H-Purin-6-yl)Amino)Ethyl)Phenol

📜2-(Benzo[b]Thiophen-3-yl)-6-Chloro-9-Isopropyl-9H-Purine,对羟基苯乙胺置于碳酸氢钠,乙酸乙酯,Sodium Sulfate-Iii,Silica Gel,正己烷,甲醇体系中,用 异丙醇 用作溶剂,化学反应 64.0H,反应生成Stemregenin1抑制剂
参考文献:Compounds That Expand Hematopoietic Stem Cells
标题:Compounds That Expand Hematopoietic Stem Cells
摘要:本发明涉及用于扩增cd34+细胞数量的化合物和组合物.该发明进一步涉及包含扩增造血干细胞(hscs)的细胞群体及其在自体或异体移植中的使用,用于治疗遗传性免疫缺陷和自身免疫性疾病以及不同的造血障碍,以重建造血细胞系和免疫系统防御.

专利信息

专利号:US-2023279438-A1
优先权日:2020-05-13
标题 :Base editing approaches for the treatment of betahemoglobinopathies
发明人：MICCIO ANNARITA; ANTONIOU PANAGIOTIS; CAVAZZANA MARINA
权利人:INSERM INSTITUT NATIONAL DE LA SANTE ET LA RECH MEDICALE; UNIV PARIS CITE; HOPITAUX PARIS ASSIST PUBLIQUE; FOND IMAGINE
摘要:The clinical history of β-hemoglobinopathies shows that the severity is mitigated by the synthesis of the fetal γ-globin in adulthood, typically associated with genetic variants the HBB cluster known as hereditary persistence of fetal hemoglobin (HPFH) mutations. The inventors identified that most of the known HPFH mutations in the γ-globin promoters (C>T, G>A, T>C or A>G) can be recapitulated using CBE- and ABE-mediatedbase-editing approaches. In particular, the inventors designed gRNAs that, when combined with CBEs or ABEs, generate HPFH mutations, and either disrupt binding sites for transcriptional repressors (-200 and -115 sites) or generate de novo DNA motifs recognized by transcriptional activators (e.g., -198 T>C, the -175 T>C and -113 A>G). It is noteworthy that a subset of the gRNAs targeting the -200 and the 115 regions are predicted to generate simultaneously HPFH mutations and also to make base changes other than HPFH mutations in or around the LRF and BCL11A binding sites, which might further reduce LRF and BCL11A occupancy. Accordingly, the present invention relates to base editing approaches for the treatment of β-hemoglobinopathies.

专利号:WO-2025017033-A1
优先权日:2023-07-17
标题:Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
发明人：MICCIO ANNARITA; CHALUMEAU ANNE; BRUSSON MÉGANE
权利人:INST NAT SANTE RECH MED; UNIV PARIS CITE; FOND IMAGINE; ASSIST PUBLIQUE HOPITAUX PARIS APHP
摘要:β-hemoglobinopathies, β-thalassemia and sickle cell disease (SCD), are monogenic diseases caused by mutations in the β-globin locus, affecting the synthesis or the structure of the adult hemoglobin (Hb). It has been shown that the severity of both SCD and β-thalassemia is lessened by the hereditary persistence of fetal hemoglobin (HbF) in adulthood (HPFH). Now the inventors exploited the capacity of prime editing to simultaneously generate multiple HPFH mutations and disrupt the BCL11A repressor BS in the -115 region of the HBG1/2 γ-globin promoters to further boost HbF expression and correct the β-thalassemia and SCD phenotypes. Specifically, the inventors selected two potent HPFH/HPFH-like mutations generating BSs for the GATA1 and KLF1 activators, that are associated with a strong γ-globin reactivation in patients and experimental models. This strategy would be very appealing for the treatment of β-hemoglobinopathies.

专利号:US-2025041330-A1
优先权日:2022-02-11
标题:Method of treating obesity by selective targeting of visceral adiposity using polycation nanomedicine
发明人：QIANG LI; LEONG KAM W; Huang Baoding; WAN QIANFEN
权利人:UNIV COLUMBIA
摘要:The method of treating obesity and targeting adipose tissue in a patient may use injection of a polycationic polymer, such as polyamidoamine (PAMAM) generation 3 (P-G3) or a PCL-g-PAMAM Denpol, that when delivered intraperitoneally, selectively targets visceral adiposity due to its high charge density. P-G3 treatment of obese mice inhibits visceral adiposity, increases energy expenditure, prevents obesity, and alleviates associated metabolic dysfunctions. The extracellular matrix of adipose tissue is enriched with glycosaminoglycans, the known biomacromolecules with the strongest negative charge. P-G3 uncouples adipocyte lipid synthesis and storage from adipocyte development, creating adipocytes with normal functions but that are deficient in hypertrophic growth. The visceral fat distribution of P-G3 is further enhanced by modifying P-G3 with cholesterol to form lipophilic nanoparticles, effective in treating obesity. This strategy provides a method to target visceral adiposity, and cationic nanomaterials useful for treating metabolic diseases or for delivery of additional treating agents.