公开(公告)号：US20210324370A1

公开(公告)日：2021-10-21

申请号：US17195625

申请日：2021-03-08

Applicant: Massachusetts Institute of Technology

Inventor： Hao Yin ,  Wen Xue ,  Daniel G. Anderson ,  Joseph R. Dorkin ,  Tyler E. Jacks

IPC: C12N15/10 ,  C12N15/11 ,  A61K48/00 ,  C12N7/00 ,  C12N15/85 ,  C12N15/86 ,  C12N15/88

Abstract: The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for deliverying such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.

公开(公告)号：US20180187186A1

公开(公告)日：2018-07-05

申请号：US15741211

申请日：2016-06-29

Applicant: Massachusetts Institute of Technology

Inventor： Hao Yin ,  Daniel G. Anderson ,  Robert Samuel Langer

IPC: C12N15/11 ,  A61K48/00 ,  C12N9/22 ,  C12N9/96 ,  C12N15/90

CPC classification number: C12N15/11 ,  A61K48/005 ,  C07H21/02 ,  C07H21/04 ,  C12N9/22 ,  C12N9/96 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/322 ,  C12N2800/80

Abstract: The disclosure relates to compositions comprising and methods for chemical modification of single guide RNA (sgRNA), tracrRNA and/or crRNA used individually or in combination with one another or Cas system components. Compositions comprising modified ribonucleic acids have been designed with chemical modification for even higher efficiency as unmodified native strand of sgRNA. Administration of modified ribonucleic acids will allow decreased immune response when administered to a subject, increased stability, increased editing efficiency and facilitated in vivo delivery of sgRNA via various delivery platforms. The disclosure also relates to methods of decreasing off-target effect of CRISPR and a CRISPR complex.

公开(公告)号：US09315472B2

公开(公告)日：2016-04-19

申请号：US14267530

申请日：2014-05-01

Applicant: Massachusetts Institute of Technology

Inventor： Yizhou Dong ,  Akinleye C. Alabi ,  Hao Yin ,  Joseph R. Dorkin ,  Delai Chen ,  Robert S. Langer ,  Daniel Griffith Anderson ,  Ahmed A. Eltoukhy

IPC: C07D251/30 ,  A61K31/53 ,  A61K31/713

CPC classification number: C07D251/30 ,  A61K31/53 ,  A61K31/713

Abstract: The present invention provides novel 1,3,5-triazinane-2,4,6-trione derivatives, such as compounds of any one of Formulae (I) and (II), and salts thereof, and methods of preparing the compounds. Also provided are compositions including a compound of the invention and an agent (e.g., an siRNA, mRNA, or plasmid DNA). The present invention also provides methods and kits using the compositions for delivering an agent to a subject (e.g., to the liver, spleen, or lung of the subject) or cell and for treating and/or preventing a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, and lung diseases.

Abstract translation: 本发明提供新的1,3,5-三氮杂环丁烷-2,4,6-三酮衍生物，如式（I）和（II）中任一项的化合物及其盐，以及制备该化合物的方法。 还提供了包含本发明化合物和试剂（例如siRNA，mRNA或质粒DNA）的组合物。 本发明还提供了使用组合物将试剂递送至受试者（例如，受试者的肝脏，脾脏或肺）或细胞的方法和试剂盒，并用于治疗和/或预防一系列疾病，例如遗传 疾病，增殖性疾病，血液疾病，神经系统疾病，肝脏疾病和肺部疾病。