公开(公告)号：US12024706B2

公开(公告)日：2024-07-02

申请号：US16988391

申请日：2020-08-07

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Julia Alterman ,  Faith Conroy ,  Edith Pfister ,  Neil Aronin ,  Ken Yamada

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/52

Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

公开(公告)号：US11827882B2

公开(公告)日：2023-11-28

申请号：US16537374

申请日：2019-08-09

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Julia Alterman ,  Faith Conroy ,  Edith Pfister ,  Neil Aronin ,  Ken Yamada

IPC: C12N15/113 ,  C12N15/85

CPC classification number: C12N15/113 ,  C12N15/85 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2310/351

Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

公开(公告)号：US10774327B2

公开(公告)日：2020-09-15

申请号：US16263200

申请日：2019-01-31

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Neil Aronin ,  Julia Alterman

IPC: C12N15/113 ,  A61K31/713 ,  A61K9/00

Abstract: This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.

公开(公告)号：US20180094264A1

公开(公告)日：2018-04-05

申请号：US15705909

申请日：2017-09-15

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Neil Aronin ,  Edith L. Pfister

IPC: C12N15/113 ,  C12N7/00 ,  A61P25/28 ,  C12N15/86

Abstract: Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

公开(公告)号：US20150232840A1

公开(公告)日：2015-08-20

申请号：US14589807

申请日：2015-01-05

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Neil Aronin ,  Phillip Zamore

IPC: C12N15/113 ,  C12Q1/68 ,  A61K31/7088

CPC classification number: C12N15/113 ,  A61K31/7088 ,  A61K47/554 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2320/32 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/158 ,  C12Q2600/178

Abstract: This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases.

Abstract translation: 本发明涉及用于治疗神经疾病的方法和组合物，更具体地涉及将iRNA试剂递送至神经细胞用于治疗神经疾病的方法。

公开(公告)号：US20140370597A1

公开(公告)日：2014-12-18

申请号：US14161266

申请日：2014-01-22

Applicant: University of Massachusetts

Inventor： Neil Aronin ,  Phillip D. Zamore

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2310/531 ,  C12N2320/30 ,  C12N2320/34

Abstract: The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.

Abstract translation: 本发明涉及发现各种功能增生疾病，特别是亨廷顿舞蹈病（HD）的有效治疗。 本发明利用RNA干扰技术（RNAi）针对编码各种增益功能突变蛋白质的基因中的多态性区域，导致功能性增益疾病的有效治疗。

公开(公告)号：US12023407B2

公开(公告)日：2024-07-02

申请号：US17154050

申请日：2021-01-21

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Reka Agnes Haraszti ,  Anastasia Khvorova ,  Neil Aronin

IPC: A61K9/127 ,  A61K31/7115 ,  A61K31/7125 ,  A61K35/17 ,  A61K38/17 ,  A61K47/28 ,  A61K47/62 ,  A61K47/69 ,  C12N15/113 ,  C12N15/86

CPC classification number: A61K9/1272 ,  A61K31/7115 ,  A61K31/7125 ,  A61K35/17 ,  A61K38/1719 ,  A61K38/1741 ,  A61K47/28 ,  A61K47/62 ,  A61K47/6917 ,  C12N15/113 ,  C12N15/86 ,  C12N2310/20 ,  C12N2310/313 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/3231

Abstract: Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.

公开(公告)号：US11773392B2

公开(公告)日：2023-10-03

申请号：US17326400

申请日：2021-05-21

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Neil Aronin ,  Edith L. Pfister

IPC: C12N15/113 ,  A61P25/28 ,  C12N7/00 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N7/00 ,  C12N15/86 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2330/51 ,  C12N2750/14121 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14171

Abstract: Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

公开(公告)号：US11767527B2

公开(公告)日：2023-09-26

申请号：US16537374

申请日：2019-08-09

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Julia Alterman ,  Faith Conroy ,  Edith Pfister ,  Neil Aronin ,  Ken Yamada

IPC: C12N15/113 ,  C12N15/85

CPC classification number: C12N15/113 ,  C12N15/85 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2310/351

Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

公开(公告)号：US11299734B2

公开(公告)日：2022-04-12

申请号：US16415826

申请日：2019-05-17

Applicant: University of Massachusetts

Inventor： Neil Aronin ,  Phillip D. Zamore

IPC: C12N15/113 ,  C12N15/11 ,  A61K48/00 ,  C07H21/02 ,  C12N5/07

Abstract: The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.