公开(公告)号：US11180751B2

公开(公告)日：2021-11-23

申请号：US15842073

申请日：2017-12-14

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  President and Fellows of Harvard College ,  Rutgers, the State University of New Jersey ,  The United States of America, as represented by the Secretary, Dept. of Health and Human Services ,  Skolkovo Institute of Science and Technology

发明人： Eugene Koonin ,  Feng Zhang ,  Yuri I. Wolf ,  Sergey Shmakov ,  Konstantin Severinov ,  Ekaterina Semenova ,  Leonid Minakhin ,  Kira S. Makarova ,  Silvana Konermann ,  Julia Joung ,  Jonathan S. Gootenberg ,  Omar O. Abudayyeh

IPC分类号： C12N15/10 ,  C12N15/11 ,  C12N9/22 ,  C12N15/82 ,  A61K48/00

摘要： The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA or RNA-targeting systems comprising a novel DNA or RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.

公开(公告)号：US20210253655A1

公开(公告)日：2021-08-19

申请号：US17251019

申请日：2019-06-14

申请人： CHILDREN'S HOSPITAL MEDICAL CENTER ,  THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY, DEPT, OF HEALTH AND HUMAN SERVICES

发明人： Douglas MILLAY ,  Dilani GAMAGE ,  Leonid CHERNOMORDIK ,  Evgenia LEIKINA

IPC分类号： C07K14/47 ,  C12N5/16

摘要： Some embodiments of the invention include polypeptides comprising a myomerger polypeptide or an extracellular myomerger polypeptide. Other embodiments of the invention include nucleic acid molecules encoding polypeptides comprising a myomerger polypeptide or an extracellular myomerger polypeptide. Other embodiments of the invention include vectors comprising the nucleic acid molecule. Yet other embodiments of the invention include methods of using a myomerger polypeptide or an extracellular myomerger polypeptide. Additional embodiments of the invention are also discussed herein.

公开(公告)号：US10905094B2

公开(公告)日：2021-02-02

申请号：US14901130

申请日：2014-06-30

申请人： The United States of America, as represented by the Secretary, Dept. of Health and Human Services

发明人： Ghadi Salem ,  John U. Dennis ,  James B. Mitchell ,  Thomas J. Pohida

IPC分类号： A01K29/00 ,  A01K1/03

摘要： The System for Continuous Observation of Rodents in Home-cage Environment (SCORHE) was developed to quantify activity levels and behavior patterns for mice housed within a home cage that, optional, may be disposed in a commercial ventilated cage rack. The SCORHE home-cage in-rack design provides daytime and night-time monitoring with the stability and consistency of the home-cage environment. The dual-video camera custom hardware design makes efficient use of space, does not require home-cage modification, and is animal facility user-friendly. In various embodiments, a software application integrates the two video streams to extract several mouse activity measures.

公开(公告)号：US20210010001A1

公开(公告)日：2021-01-14

申请号：US17024100

申请日：2020-09-17

申请人： Sarepta Therapeutics, Inc. ,  THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES ,  University of Maryland ,  The Progeria Research Foundation

发明人： Michael R. Erdos ,  Francis S. Collins ,  Kan Cao ,  Ryszard Kole ,  Richard Keith Bestwick ,  Leslie B. Gordon

IPC分类号： C12N15/113 ,  A61P43/00 ,  C07K7/06

摘要： Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

公开(公告)号：US10767184B2

公开(公告)日：2020-09-08

申请号：US15692473

申请日：2017-08-31

申请人： The United States of America, as represented by the Secretary, Department of Health and Human Services

发明人： James N. Kochenderfer

IPC分类号： C12N15/62 ,  C07K14/705 ,  C07K14/725 ,  C07K16/28 ,  C07K16/18 ,  A61K48/00 ,  A61K39/00

摘要： The invention provides an isolated and purified nucleic acid sequence encoding a chimeric antigen receptor (CAR) directed against B-cell Maturation Antigen (BCMA). The invention also provides host cells, such as T-cells or natural killer (NK) cells, expressing the CAR and methods for destroying multiple myeloma cells.

公开(公告)号：US20200147010A1

公开(公告)日：2020-05-14

申请号：US16594827

申请日：2019-10-07

申请人： MESHABERASE, LLC ,  THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES

发明人： Benjamin RUBIN ,  Raj PURI ,  Bharat JOSHI

IPC分类号： A61K31/145 ,  A61K9/48 ,  A61K9/28 ,  A61K9/00 ,  A61K45/06

摘要： The present Disclosure is directed to methods for inhibiting or suppressing metastasis of a tumor in a mammalian subject using a cysteamine product, e.g., cysteamine or cystamine or a derivative thereof. Also described herein is a method for treating pancreatic cancer in a mammalian subject by administering a cysteamine product described herein.

公开(公告)号：US20200095306A1

公开(公告)日：2020-03-26

申请号：US16443415

申请日：2019-06-17

申请人： The United States of America, as represented by the Secretary, Dept. of Health and Human Services ,  Washington University

发明人： Jeffrey S. Isenberg ,  David D. Roberts ,  William A. Frazier

IPC分类号： C07K16/18 ,  A61K48/00 ,  G01N33/68 ,  C07K14/78 ,  A61K39/395 ,  A61K45/06 ,  B01J21/06 ,  B01J21/12 ,  B01J23/00 ,  B01J23/30 ,  B01J27/188 ,  B01J37/02 ,  C07C45/00 ,  C07C45/52 ,  C07C51/235 ,  C07C51/25 ,  C07C253/26

摘要： Provided herein are compositions for preventing, ameliorating, and/or reducing tissue ischemia and/or tissue damage due to ischemia, increasing blood vessel diameter, blood flow and tissue perfusion in the presence of vascular disease including peripheral vascular disease, atherosclerotic vascular disease, coronary artery disease, stroke and influencing other conditions, by suppressing CD47 and/or blocking TSP1 and/or CD47 activity or interaction. Influencing the interaction of CD47-TSP1 in blood vessels allows for control of blood vessel diameter and blood flow, and permits modification of blood pressure and cardiac function. Under conditions of decreased blood flow, for instance through injury or atherosclerosis, blocking TSP1-CD47 interaction allows blood vessels to dilate and increases blood flow, tissue perfusion and tissue survival.

公开(公告)号：US20200030435A1

公开(公告)日：2020-01-30

申请号：US16596175

申请日：2019-10-08

申请人： The United States of America, as represented by the Secretary, Dept. of Health and Human Services ,  The Government of the United States, as Represented by The Secretary of The Army

发明人： Alexander G. Pletnev ,  Joseph R. Putnak ,  Robert M. Chanock ,  Brian R. Murphy ,  Stephen S. Whitehead ,  Joseph E. Blaney

IPC分类号： A61K39/12 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12Q1/6888

摘要： The present invention relates to attenuated, immunogenic West Nile virus chimeras built on a dengue virus backbone for the production of immunogenic, live, attenuated West Nile virus vaccines.

公开(公告)号：US10350306B2

公开(公告)日：2019-07-16

申请号：US15876821

申请日：2018-01-22

申请人： The United States of America, as represented by THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES

发明人： Paul Albert Sieving ,  Ronald Avery Bush ,  Peter C. Colosi ,  Yong Zeng

IPC分类号： A61K48/00 ,  A61P27/02 ,  A61K38/17 ,  A61K9/00 ,  A61K45/06 ,  A61K31/592 ,  A61K31/593 ,  C12N15/86 ,  A61K33/06 ,  A61K31/5377 ,  A61K31/573

摘要： Expression vectors and therapeutic methods of using such vectors in the treatment of diseases of the eye resulting from failure to produce a specific protein in the eye, or the production of a non-functional protein in the eye.

公开(公告)号：US20190194260A1

公开(公告)日：2019-06-27

申请号：US16083652

申请日：2017-03-11

申请人： The United States of America, as represented by the Secretary, Dept. of Health and Human Services ,  The Johns Hopkins University

发明人： Stephen S. Whitehead ,  Sara E. Woodson ,  Anna P. Durbin ,  Alexander G. Pletnev ,  Konstantin A. Tsetsarkin

IPC分类号： C07K14/005 ,  A61K39/12

CPC分类号： C07K14/005 ,  A61K39/12 ,  A61K2039/5254 ,  A61K2039/5256 ,  C12N2770/24122 ,  C12N2770/24134 ,  C12N2770/24144 ,  C12N2770/24162 ,  Y02A50/386 ,  Y02A50/388 ,  Y02A50/39 ,  Y02A50/392 ,  Y02A50/394 ,  Y02A50/396

摘要： The present disclosure relates to attenuated Zika viruses and vaccines, attenuated chimeric Zika viruses and vaccines, and to multivalent immunogenic compositions comprising Zika vaccines and vaccines to other flaviviruses. The chimeric Zika viruses includes a first nucleotide sequence encoding at least one structural protein from a Zika virus (ZIKV), a second nucleotide sequence encoding at least one nonstructural protein from a first flavivirus, and a third nucleotide sequence of a 3′ untranslated region from a second flavivirus. The multivalent immunogenic compositions comprise an attenuated ZIKV vaccine or an attenuated chimeric ZIKV vaccine (or their combination) together with one or more of a first attenuated virus that is immunogenic against dengue serotype 1, a second attenuated virus that is immunogenic against dengue serotype 2, a third attenuated virus that is immunogenic against dengue serotype 3, and a fourth attenuated virus that is immunogenic against dengue serotype 4. The present disclosure further relates to methods of inducing immune responses, as well as preventing or treating ZIKV infections, and in certain embodiments, the combined prevention or treatment of ZIKV and another flavivirus, e.g., dengue virus.