公开(公告)号：US20150232883A1

公开(公告)日：2015-08-20

申请号：US14705719

申请日：2015-05-06

Applicant: THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： James Dahlman ,  Randall Jeffrey Platt ,  Daniel G. Anderson ,  Robert S. Langer ,  Feng Zhang

IPC: C12N15/90

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/102 ,  C12N15/63

Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery particle formulations and/or systems comprising one or more components of a CRISPR-Cas system, which are means for targeting sites for delivery. The delivery particle formulations of the invention are preferably nanoparticle delivery formulations and/or systems. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Abstract translation: 本发明提供了用于操纵靶序列的序列和/或活性的系统，方法和组合物的递送，工程和优化。 提供的是包含CRISPR-Cas系统的一个或多个组分的递送颗粒制剂和/或系统，其是用于靶向用于递送的位点的装置。 本发明的递送颗粒制剂优选为纳米颗粒递送制剂和/或系统。 还提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及用于设计和使用这些载体的方法。 还提供了在真核细胞中引导CRISPR复合物形成的方法，以确保增强的靶标识别和避免毒性的特异性，以及编辑或修饰感兴趣的基因组基因座中的靶位点以改变或改善疾病或病症的状态。

公开(公告)号：US20150025005A1

公开(公告)日：2015-01-22

申请号：US14379477

申请日：2013-02-19

Applicant: Massachusetts Institute of Technology

Inventor： Robert S. Langer ,  Daniel G. Anderson ,  Zhen Gu ,  Alex Arthur Aimetti

IPC: C07K7/06 ,  A61K9/00 ,  A61K47/42

CPC classification number: C07K7/06 ,  A61K9/0019 ,  A61K9/06 ,  A61K38/00 ,  A61K38/28 ,  A61K47/42

Abstract: A glucose binding amphiphilic peptide hydrogel insulin delivery system that is responsive to glucose concentrations under physiological conditions is provided. Insulin is encapsulated in a glucose binding hydrogel, made from self-assembling amphiphilic peptides including a hydrophobic domain including a beta sheet forming region coupled to a charged hydrophilic domain modified to contain a glucose binding segment. The formulations are designed to release insulin as a function of blood glucose level, maintaining the patients' blood glucose level in an optimum range and avoiding both hyper- and hypoglycemia.

Abstract translation: 提供了在生理条件下对葡萄糖浓度有反应的葡萄糖结合两亲肽水凝胶胰岛素递送系统。 胰岛素包封在葡萄糖结合水凝胶中，由自组装两亲肽制成，包括疏水结构域，其包括与修饰成含有葡萄糖结合片段的带电亲水结构域连接的β片形成区。 该制剂被设计为释放胰岛素作为血糖水平的函数，将患者的血糖水平维持在最佳范围内并避免高血糖和低血糖。

公开(公告)号：US20140271843A1

公开(公告)日：2014-09-18

申请号：US13831250

申请日：2013-03-14

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： Minglin Ma ,  Daniel G. Anderson ,  Robert S. Langer ,  Omid Veiseh ,  Arturo Jose Vegas ,  Joshua Charles Doloff

IPC: A61K9/48 ,  A61K35/39

CPC classification number: A61K35/39 ,  A61K9/0024 ,  A61K9/5036 ,  A61K9/5078 ,  A61K9/5089 ,  A61K2035/126 ,  A61K2035/128 ,  B01J13/046

Abstract: Biocompatible hydrogel capsules encapsulating mammalian cells having a diameter of greater than 1 mm, and optionally a cell free core, are disclosed which have reduced fibrotic overgrowth after implantation in a subject. Methods of treating a disease in a subject are also disclosed that involve administering a therapeutically effective amount of the disclosed encapsulated cells to the subject.

Abstract translation: 公开了包封直径大于1mm的哺乳动物细胞和任选的无细胞核的生物相容性水凝胶胶囊，其在植入受试者后具有减少的纤维化过度生长。 还公开了治疗受试者疾病的方法，其涉及向受试者施用治疗有效量的所公开的封闭细胞。

公开(公告)号：US20140125196A1

公开(公告)日：2014-05-08

申请号：US14071482

申请日：2013-11-04

Applicant: Massachusetts Institute of Technology

Inventor： Mingming Ma ,  Liang Guo ,  Daniel G. Anderson ,  Omid C. Farokhzad ,  Robert S. Langer

IPC: H01L41/193 ,  H01L41/37

CPC classification number: H01L41/193 ,  C08G2210/00 ,  C08G2261/3221 ,  C08G2261/44 ,  C08J3/075 ,  C08J2371/02 ,  C08J2465/00 ,  C08K3/38 ,  C08K5/053 ,  C08L65/00 ,  C08L71/02 ,  H01L41/37

Abstract: Water-responsive composite materials are provided containing a polymeric matrix and a water-responsive gel integrated into the polymeric matrix. The water-responsive gel can include a polyol or an alkoxylated polyol crosslinked by reversibly hydrolysable bonds, such as borate ester bonds. The polymeric matrix can include conjugated polymers such as poly(pyrrole) containing polymers. The composite material is capable of rapid actuation in the presence of a water gradient and can exhibit power densities greater than 1 W/kg. Methods of making water-responsive composite materials are provided, including by electropolymerization. Devices containing water-responsive composite materials are provided for sensing, locomotion, and power generation.

Abstract translation: 提供了水反应性复合材料，其包含聚合物基质和整合到聚合物基质中的水响应性凝胶。 水反应性凝胶可以包括通过可逆水解的键如硼酸酯键交联的多元醇或烷氧基化多元醇。 聚合物基质可以包括共轭聚合物，例如含聚（吡咯）的聚合物。 复合材料能够在水梯度的存在下快速致动，并且可以显示大于1W / kg的功率密度。 提供制备水响应复合材料的方法，包括通过电聚合。 提供含水响应复合材料的设备用于感测，运动和发电。