公开(公告)号：US11760999B2

公开(公告)日：2023-09-19

申请号：US17174452

申请日：2021-02-12

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Robert H. Brown, Jr.

IPC: C07H21/04 ,  C12N15/113 ,  C12N15/11 ,  C12N15/86

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/86 ,  C12Y115/01001 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2750/14143

Abstract: The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

公开(公告)号：US11739330B2

公开(公告)日：2023-08-29

申请号：US16649164

申请日：2018-09-21

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Robert H. Brown

IPC: C12N15/113 ,  C12N15/86 ,  C12N7/00 ,  A61K35/76 ,  A61K48/00

CPC classification number: C12N15/1137 ,  A61K35/76 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00 ,  C12N2750/14043

Abstract: In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.

公开(公告)号：US20230089312A1

公开(公告)日：2023-03-23

申请号：US17801917

申请日：2021-02-24

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Neil Aronin

IPC: C12N15/63 ,  C12N15/86 ,  C12N9/12

Abstract: Aspects of the disclosure relate to compositions and methods for regulation of transgene (e.g., miRNAs, shRNAs or coding sequences) expression from viral vectors. In some embodiments, the disclosure provides expression constructs comprising a viral vector encoding one or more transgenes, the expression of which is regulated by a rapamycin/rapalog-based system.

公开(公告)号：US20200248187A1

公开(公告)日：2020-08-06

申请号：US16649164

申请日：2018-09-21

Applicant: University of Massachusetts One Beacon Street

Inventor： Christian Mueller ,  Robert H. Brown

IPC: C12N15/113 ,  C12N15/86 ,  C12N7/00 ,  A61K35/76

Abstract: In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.

公开(公告)号：US20200032256A1

公开(公告)日：2020-01-30

申请号：US16295621

申请日：2019-03-07

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Robert H. Brown, JR.

IPC: C12N15/113 ,  C12N15/11 ,  C12N15/86

Abstract: The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

公开(公告)号：US20190211327A1

公开(公告)日：2019-07-11

申请号：US16059121

申请日：2018-08-09

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Christian Mueller ,  Phillip D. Zamore

IPC: C12N15/113 ,  A61K48/00 ,  A61K38/57 ,  A61K31/713 ,  C07K14/81 ,  A61K35/12 ,  C12N7/00 ,  C12N15/86 ,  C12N15/67

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K35/12 ,  A61K38/57 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/8125 ,  C12N7/00 ,  C12N15/111 ,  C12N15/67 ,  C12N15/86 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2320/31 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

公开(公告)号：US20240084303A1

公开(公告)日：2024-03-14

申请号：US18451147

申请日：2023-08-17

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 (111 1) and methods of treating Huntington's disease using the same.

公开(公告)号：US20240076668A1

公开(公告)日：2024-03-07

申请号：US18362190

申请日：2023-07-31

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Robert H. Brown, JR.

IPC: C12N15/113 ,  C12N15/11 ,  C12N15/86

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/86 ,  C12Y115/01001 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2750/14143

Abstract: The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

公开(公告)号：US20230416757A1

公开(公告)日：2023-12-28

申请号：US18344926

申请日：2023-06-30

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Robert H. Brown, JR.

IPC: C12N15/113 ,  A61K35/76 ,  C12N7/00 ,  C12N15/86

CPC classification number: C12N15/1137 ,  A61K35/76 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00

Abstract: In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.

公开(公告)号：US20220033824A1

公开(公告)日：2022-02-03

申请号：US17297521

申请日：2019-11-27

Applicant: University of Massachusetts

Inventor： Christian Mueller ,  Gabriela Toro Cabrera ,  Robert H. Brown, JR.

IPC: C12N15/113 ,  C12N15/86 ,  C12N15/10 ,  A61K35/761

Abstract: The disclosure provides, in some aspects, compositions (e.g., isolated nucleic acids and rAAVs) comprising a transgene which encodes at least one inhibitory nucleic acid which decreases expression of a gene encoding a serine palmitoyltransferase protein (e.g., SPTLC1). In some aspects, the disclosure relates to methods of treating hereditary sensory and autonomic neuropathy 1 (HSAN1) by administering the compositions described by the disclosure to a cell or subject.