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SYNTHETIC SINGLE STRANDED NUCLEIC ACID COMPOSITIONS AND METHODS THEREOF

Resumen de: AU2023406947A1

The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.

COMPOSITIONS AND METHODS FOR MAKING AND USING POLYMER-COATED NANOCAPSULES FOR TARGETED PHARMACEUTICAL AGENT DELIVERY

Resumen de: WO2024118638A2

Embodiments of the present disclosure provide novel compositions and methods for making and using polymer-coated nanocapsules. In certain embodiments, compositions and methods are disclosed for embedding at least one agent in a liquid fatty acid composition to form an inner core of the polymer-coated nanocapsule and coating the at least one agent-containing liquid fatty acid composition inner core with polymer to form at least one coating layer of polymer that further includes at least one positively charged surfactant (e.g., cationic surfactant), forming polymer-coated nanocapsules. In certain embodiments, the at least one positively charged surfactant binds to at least one targeting agent for directed use of the polymer-coated nanocapsules.

TARGETING GPR158 (MGLYR) WITH NANOBODIES FOR THERAPEUTIC BENEFITS

Resumen de: WO2024118636A1

The invention provides antibodies that specifically bind GPR158 and inhibit GAP activity of GPR158 via RGS7/ Gβ5. The antibodies are useful in the diagnosis and treatment of affective disorders, mood disorders, and brain disorders.

VACCINES CONTAINING NOVEL NANOPARTICLE SCAFFOLDS

Resumen de: AU2023399881A1

The present invention provides novel engineered nanoparticle scaffold sequences that are derived from the 13-01 protein. Relative to the known 13-01 protein or variants thereof, the novel 13-01 derived scaffold sequences of the invention contain an extended N-terminal helix. Also provided in the invention are vaccine constructs that contain various immunogenic proteins displayed on the novel nanoparticle scaffold sequences described herein. The vaccine constructs of the invention include, e.g., nanoparticles displaying tandem repeats of influenza M2e proteins or HCV E2 core proteins.

COMPOSITIONS AND METHODS FOR TREATING HUMAN PAPILLOMAVIRUS INFECTIONS

Resumen de: US2024342163A1

The invention provides compositions, compounds, formulations, and methods for treating HPV infections including pre-malignant infections and cancer. Compounds that covalently bind to the HPV E6 protein are disclosed.

TARGETING NANOSCALE PARTICLE, TARGETING CELL, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: EP4628071A1

The invention discloses a targeted nanoscale particle, a targeted cell, a preparation method therefor, and use thereof. The targeted nanoscale particle is bound to the outer surface of the targeted cell, and is composed of a plurality of proteins interconnected via a first binding site. The targeted nanoscale particle further comprises a second binding site, and is bound to the outer surface of a target cell via the second binding site. In an exemplary embodiment, the targeted nanoscale particle can promote the interaction between the two types of cells by simultaneously binding to a chimeric antigen receptor T cell and a leukemia cell, thereby promoting the recognition and killing of the leukemia cell by the chimeric antigen receptor T cell. In addition, the internal cavities of the proteins in the targeted nanoscale particle provide space for loading of a chemotherapeutic drug, thus realizing the combination therapy of the chimeric antigen receptor T cell and other therapies while loading the drug.

アルツハイマー病を処置するための新たな合成薬

Resumen de: WO2024058196A1

The present invention aims to provide a novel agent for treating Alzheimer's disease, a method for treating Alzheimer's disease, a method for screening for a candidate substance for a therapeutic drug for Alzheimer's disease, and the like. The present invention is a prophylactic and/or therapeutic agent for Alzheimer's disease comprising a peptide corresponding to dynamin 1. The peptide preferably corresponds to dynamin 1-pleckstrin-homology domain or dynamin 1-proline rich domain. In addition, the peptide is preferably encapsulated in nano-particles or linked to a peptide sequence that improve delivery of the peptide into the brain.

一种外泌体包覆的共负载金纳米颗粒和抗生素的树状大分子纳米凝胶及其制备方法和应用

Resumen de: CN120732815A

本发明涉及一种外泌体包覆的共负载金纳米颗粒和抗生素的树状大分子纳米凝胶及其制备方法和应用，以树状大分子纳米凝胶为载体，载体内部共负载金纳米颗粒和药物，载体表面包裹外泌体。本发明操作条件简单，易于纯化，可改善抗生素的生物利用度并实现药物在病灶部位的靶向递送和响应性释放，在肺结核治疗中拥有良好的应用前景。

一种脂质纳米颗粒及其应用

Resumen de: CN120732814A

本发明涉及一种脂质纳米颗粒及其应用。所述脂质纳米颗粒包括：阳离子脂质、阴离子脂质DOPS（1,2‑二油酰基‑sn‑甘油‑3‑磷脂酰‑L‑丝氨酸）、其他脂质和靶向抗体。本发明经过研究发现，掺杂阴离子脂质DOPS并间接偶联靶向抗体可以显著提高纳米颗粒对脾脏T细胞的转染效率。本发明进一步将编码靶向肿瘤血管内皮细胞和肿瘤细胞的CAR蛋白的核酸装载在上述脂质纳米颗粒中并用于肿瘤治疗，结果上述治疗显著促进了免疫细胞的瘤内浸润，显著抑制多种实体瘤进展，为CAR‑T疗法在实体瘤中的有效应用提供了新思路、新手段。

一种氨基酸阳离子脂质

Resumen de: CN117460710A

The invention provides a novel amino acid cationic lipid, the structure of which is shown in a general formula (1), and the definition of each symbol is consistent with that described in the specification. The amino acid cationic lipid is a pharmaceutically acceptable, biodegradable or high-biocompatibility lipid, and has the advantages of low toxicity, low immunogenicity and high biocompatibility. The amino acid or the amino acid derivative used in the preparation process is simple and easy to obtain, can be naturally obtained or simply synthesized, and has the advantages of simplicity, convenience, safety and production cost saving. Degradable groups can further be contained between amino acid residues and lipophilic tail chains of the novel amino acid cationic lipid, due to existence of the degradable groups, lipid nanoparticles LNP prepared from the novel amino acid cationic lipid can be degraded in vivo in good time, and the problems that in the prior art, LNP prepared from lipid which cannot be degraded can be stored in vivo, the endosome environment is acidified, and the lipid nanoparticles LNP cannot be degraded in vivo are solved. Endosome escape of drug molecules (such as nucleic acid) is blocked, and the problem that drugs delivered into cells cannot play a role is solved. # imgabs0 #

经由穿膜肽进行非共价修饰脂质纳米颗粒缀合物及用途

Resumen de: CN120737148A

本发明提供了一种多肽衍生物，其具有结构式CnH2n+1CONH‑CPP‑COOH，其中，n=11，13，15，17；CPP为穿膜肽。本发明还提供了该多肽衍生物的制备方法和与脂质纳米颗粒缀合的用途。本发明通过加入在LNP配方中加入基于CPP的阳离子两亲性分子，由可电离脂质负责mRNA的内体释放而CPP促进LNP与细胞膜的结合，通过调整各组分组成比例以及电荷，显著提升了LNP在体内外的递送效率，经由TAT序列修饰LNP可使得肝脏体内递送效率提升20倍。

一种具有肿瘤能量代谢调控功能的自组装基因纳米药物及其制备方法和应用

Resumen de: CN120733055A

本发明公开了一种具有肿瘤能量代谢调控功能的自组装基因纳米药物及其制备方法和应用，属于纳米材料技术领域。本发明通过将光敏剂与阳离子聚合物偶联，形成基因递送载体，该复合物在特定光照条件下能够增强肿瘤细胞的凋亡效果；通过将光敏剂与siRNA结合，实现了多重治疗机制的协同作用，不仅能精准靶向肿瘤细胞并干预其能量代谢，还能在光照下增强细胞死亡效应，显著提高治疗效果。本发明通过靶向肿瘤细胞中高表达的与能量代谢相关的基因，并结合光动力疗法，解决了现有技术在肿瘤能量代谢干预方面的不足，有望在未来成为靶向细胞器的新型治疗手段，可用于抗癌、抗菌、抗炎等药物治疗及示踪。

一种基于Mn-DNA纳米疫苗的肿瘤免疫治疗方法

Resumen de: CN120733013A

本发明涉及一种基于Mn‑DNA纳米疫苗的肿瘤免疫治疗方法。本发明提供一种用于肿瘤免疫治疗的纳米颗粒，其包含金属离子Mn2+、外源dsDNA、抗原肽和打靶肽。本发明还提供制备所述纳米颗粒的方法、包含所述纳米颗粒的疫苗或药物组合物以及它们用于肿瘤免疫治疗的用途等。本发明的纳米颗粒能够被精准递送到抗原提呈细胞，促进抗原提呈细胞成熟，增强抗原递呈能力，显著增强肿瘤靶向性和抗肿瘤活性。本发明所用原料在体内均可降解，生物安全性高。

编码治疗性多肽的核酸及包含所述核酸的脂质纳米颗粒组合物

Resumen de: WO2024199114A1

Provided are lipid nanoparticle compositions comprising nucleic acids encoding RSV antigenic polypeptides. Also provided are novel antigenic RSV-F polypeptides as well as nucleic acids encoding the antigenic RSV-F polypeptides.

一种氨基酸阳离子脂质

Resumen de: CN117460710A

The invention provides a novel amino acid cationic lipid, the structure of which is shown in a general formula (1), and the definition of each symbol is consistent with that described in the specification. The amino acid cationic lipid is a pharmaceutically acceptable, biodegradable or high-biocompatibility lipid, and has the advantages of low toxicity, low immunogenicity and high biocompatibility. The amino acid or the amino acid derivative used in the preparation process is simple and easy to obtain, can be naturally obtained or simply synthesized, and has the advantages of simplicity, convenience, safety and production cost saving. Degradable groups can further be contained between amino acid residues and lipophilic tail chains of the novel amino acid cationic lipid, due to existence of the degradable groups, lipid nanoparticles LNP prepared from the novel amino acid cationic lipid can be degraded in vivo in good time, and the problems that in the prior art, LNP prepared from lipid which cannot be degraded can be stored in vivo, the endosome environment is acidified, and the lipid nanoparticles LNP cannot be degraded in vivo are solved. Endosome escape of drug molecules (such as nucleic acid) is blocked, and the problem that drugs delivered into cells cannot play a role is solved. # imgabs0 #

一种用于治疗急性呼吸窘迫综合征的仿胞葬纳米制剂

Resumen de: CN120732785A

本发明公开了一种用于治疗急性呼吸窘迫综合征的仿胞葬纳米制剂，属于生物医药技术领域。本发明的仿胞葬纳米制剂包括活性成分、脂质体磷脂双分子层和靶头，所述活性成分由ROS清除药物和抗炎药物组成。本发明创新性地运用仿胞葬作用实现纳米制剂的高效精准靶向，在脂质体表面修饰凋亡细胞标志物，通过释放“eat me”信号，模拟巨噬细胞清除凋亡细胞的天然生物学过程被肺泡巨噬细胞识别并吞噬，实现高效特异性靶向，同时炎症部位巨噬细胞PS受体表达上调进一步增强了靶向效果，为急性呼吸窘迫综合征药物制剂高效递送及病理微环境的重塑提供了一种新的途径。

一种氨基酸阳离子脂质

Resumen de: CN117460710A

The invention provides a novel amino acid cationic lipid, the structure of which is shown in a general formula (1), and the definition of each symbol is consistent with that described in the specification. The amino acid cationic lipid is a pharmaceutically acceptable, biodegradable or high-biocompatibility lipid, and has the advantages of low toxicity, low immunogenicity and high biocompatibility. The amino acid or the amino acid derivative used in the preparation process is simple and easy to obtain, can be naturally obtained or simply synthesized, and has the advantages of simplicity, convenience, safety and production cost saving. Degradable groups can further be contained between amino acid residues and lipophilic tail chains of the novel amino acid cationic lipid, due to existence of the degradable groups, lipid nanoparticles LNP prepared from the novel amino acid cationic lipid can be degraded in vivo in good time, and the problems that in the prior art, LNP prepared from lipid which cannot be degraded can be stored in vivo, the endosome environment is acidified, and the lipid nanoparticles LNP cannot be degraded in vivo are solved. Endosome escape of drug molecules (such as nucleic acid) is blocked, and the problem that drugs delivered into cells cannot play a role is solved. # imgabs0 #

一种用于金黄色葡萄球菌感染检测灭活的诊疗探针

Resumen de: CN120733070A

本发明公开了一种用于金黄色葡萄球菌感染检测灭活的诊疗探针，构建方法包括：S1：双波长激发量子点的制备；S2：量子点@ZIF‑8的制备，在量子点表面进行PSS修饰，后在ZIF‑8包覆过程中在1小时内匀速缓慢滴加2‑甲基咪唑溶液用以控制纳米颗粒的成核速率，并且加入PVP稳定纳米粒子的形成；S3：量子点@ZIF‑8@Ber的制备，在合成过程中将小檗碱加入溶剂中，采用一锅法直接合成得到负载小檗碱的量子点@ZIF‑8材料。本发明利用夹心法对金黄色葡萄球菌进行检测，结果通过荧光检测仪反馈，并且可以连接手机进行智能化监测，简便快捷；利用抗原抗体反应，靶向捕获金黄色葡萄球菌，其余细菌的影响很小。

一种生物合成虾青素的马氏副球菌及其色素提取物和应用

Resumen de: CN120738011A

本发明公开了一种生物合成虾青素的马氏副球菌及其色素提取物和应用。该马氏副球菌（Paracoccus marcusii）IHA069于2022年7月15日保藏于中国普通微生物菌种保藏中心，保藏编号为CGMCC No.25302，16s rDNA序列如SEQ ID No.1所示。通过发酵培养基培养48h后，离心并采用无水乙醇萃取菌体得到色素提取物，虾青素在色素提取物中的比例达到40%。马氏副球色素提取物为主料，玉米淀粉、吐温‑80、氯化钙水为配料，调和喷雾干燥得到具有抗氧化效果的纳米颗粒。相较于IHA034，IHA069强化了提高了虾青素产量便于工业生产，提高了在不同动物中使用的可行性。本发明采用纳米颗粒包埋技术提高了色素提取物中虾青素的保留率，提高了保存温度和有效时间。

新型细胞因子组合mRNA疫苗佐剂及其应用

Resumen de: CN120733023A

本发明提供了一种新型细胞因子组合mRNA疫苗佐剂及其应用，所述疫苗佐剂包括可电离脂质、胆固醇、磷脂、PEG衍生物，以及含有编码GM‑CSF和IL‑21基因序列的mRNA；其中，GM‑CSF和IL‑21的氨基酸序列分别如SEQ ID NO:1、2所示。将本发明所述新型细胞因子组合mRNA疫苗佐剂应用于疫苗的制备，组合的细胞因子在注射部位对DC的招募和调控促进了LNP递送和表达引发了强烈的抗肿瘤效应。具体的，通过协同作用通过对注射部位免疫微环境的建立提升了抗原被表达、识别和呈递并且促进了抗原的交叉提呈，DC细胞的激活和淋巴结的浸润促进了T细胞的活化，为肿瘤免疫治疗建立了强大了抗肿瘤效应和免疫记忆。

一种可精准质控的干细胞纳米囊泡及其应用

Resumen de: CN119530147A

The invention relates to a stem cell bioactive nano-vesicle with efficient inflammation regulation and tissue repair effects, the average particle size of the nano-vesicle is 100-350nm, the polydispersity index (PDI) is 0.05-0.40, and the expression rate of phosphatidylserine (PS) on the surface of the nano-vesicle is greater than 40%. According to the preparation method, stem cells are subjected to apoptosis induction, and then a programmed serial extrusion process is adopted to prepare the nano-vesicles. The nano vesicles are moderate in particle size, uniform in distribution and high in production efficiency, the preparation process is easy to operate, the quality is stable and controllable, and the nano vesicles have excellent technical effects in the aspects of repair and regeneration of inflammation injury type tissues and organs.

一种负载葛花异黄酮的纳米颗粒及其制备方法和应用

Resumen de: CN120732816A

本发明公开了一种负载葛花异黄酮的纳米颗粒及其制备方法和应用，属于复合纳米颗粒技术领域。本发明的负载葛花异黄酮的纳米颗粒，包括纳米颗粒中心和多糖外壳；纳米颗粒中心为包覆葛花异黄酮的酒糟醇溶蛋白，多糖外壳由内至外依次为硫酸葡聚糖外壳和壳聚糖外壳。其制备方法包括以下步骤：以碳酸钠为牺牲模版制备负载葛花异黄酮的单层纳米颗粒；通过调节溶液pH，使纳米粒子带正电，以静电沉积效应涂覆负电性的硫酸葡聚糖外壳；然后与壳聚糖溶液混合，进一步涂覆正电性的壳聚糖外壳，形成层层自组装结构。本发明的负载葛花异黄酮的纳米颗粒在体外模拟消化环境中释放率更高，清除自由基活性更强，为葛花异黄酮的递送提供了更高效的技术。

一种罗汉果外泌体样纳米颗粒及其制备方法和在改善肠道健康产品中的应用

Resumen de: CN120732915A

本发明公开一种罗汉果外泌体样纳米颗粒及其制备方法和在改善肠道健康产品中的应用，将罗汉果果肉与预冷PBS匀浆，离心取上清液，经超速离心获得粗提液；通过羧基化磁珠偶联磷脂酰丝氨酸靶向多肽，构建免疫磁珠层析柱，特异性捕获并纯化外泌体样纳米颗粒；所得纳米颗粒具有优异的生物相容性和安全性，可显著调节肠道菌群平衡、增强肠道屏障功能、抑制肠道炎症；本发明为肠道疾病治疗及功能性食品开发提供了天然、高效的新方案，具有广阔的医药和食品应用前景。

一种靶向放疗增敏剂、仿生纳米递送系统及其制备方法和应用

Resumen de: CN120733052A

本申请涉及生物医用纳米材料技术领域，公开了一种靶向放疗增敏剂、仿生纳米递送系统及其制备方法和应用。靶向放疗增敏剂的制备方法包括：将三苯基磷和多肽缩合剂溶于无水DMF中，搅拌均匀；再加入SH‑PEG2000‑NH2溶液进行反应，得到SH‑PEG2000‑TPP；将氯金酸溶液加热回流，再加入柠檬酸钠溶液，反应后得到AuNPs分散液；将AuNPs分散液、SH‑PEG2000‑NH2和SH‑PEG2000‑TPP混合反应，得到AuNP‑PEG2000‑TPP纳米粒子，即靶向放疗增敏剂。本申请的靶向放疗增敏剂进入肿瘤线粒体后，经过X射线照射，能够有效吸收射线能量，并转化为热能和电子激发能，产生活性氧(ROS)，进而激活炎性小体和caspase‑1，随后产生N端GSDMD蛋白片段，启动焦亡过程，发挥较强的放疗增敏作用；焦亡过程中释放IL‑1β和IL‑18等促炎细胞因子，进一步扩大局部的免疫反应。

てんかん治療のためのプレプロダイノルフィンバリアントの発現のための遺伝子治療ベクター

Nº publicación: JP2025532800A 03/10/2025

Solicitante:

シャリテ－ウニベルジテーツメディツィンベルリン

Resumen de: CN119968387A

The subject matter of the present invention is a delivery vector comprising a DNA sequence encoding pro-predynorphin or a variant of pro-predynorphin and wherein said delivery vector drives the expression of pro-predynorphin in a target cell and wherein said delivery vector comprising said DNA sequence is capable of releasing predynorphin or a variant of predynorphin from a target cell as needed, and wherein the pro-pro-peptide is pro-pro-peptide or a pro-pro-peptide variant and wherein the pro-pro-peptide comprises a signal peptide wherein the signal peptide is an N-terminal extension of a neonatal polypeptide chain and wherein the signal peptide mediates a protein targeting an endoplasmic network lumen, and wherein the pro-peptidogen comprises an N-terminal pro-peptidogen fragment on the C-terminus of the signal peptide and wherein the N-terminal pro-peptidogen fragment (i) comprises a sorting motif comprising elements DL and EXyL, in particular a sorting motif consisting of the amino acid sequence DLXxEXyL, or comprising ii) a sorting motif of pro-neuropeptidic or protein sorted into a dense core macrovesicle, except for pro-prodynorphin as defined herein, where in certain embodiments, x is an integer from 1 to 20, y is an integer from 1 to 10, and each instance of X may independently be any amino acid, such as any amino acid, or any amino acid, such as any amino acid, or any amino acid, such as any amino acid, or any amino acid, such as any amino acid, or any amino acid,