公开(公告)号：US10711274B2

公开(公告)日：2020-07-14

申请号：US16364126

申请日：2019-03-25

申请人： University of Massachusetts

发明人： Christian Mueller ,  Robert H. Brown, Jr.

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

摘要： 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：US20230295629A1

公开(公告)日：2023-09-21

申请号：US18178206

申请日：2023-03-03

申请人： UNIVERSITY OF MASSACHUSETTS

发明人： Robert H. Brown, Jr. ,  Jonathan K. Watts ,  Helene Tran ,  Michael Moazami

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/3341 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/315

摘要： The present disclosure provides antisense compounds, methods, and compositions for silencing C9ORF72 transcripts. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with C9ORF72 in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with C9ORF72.

公开(公告)号：US10851375B2

公开(公告)日：2020-12-01

申请号：US16403948

申请日：2019-05-06

申请人： University of Massachusetts

发明人： Christian Mueller ,  Robert H. Brown, Jr.

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

摘要： 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：US11629347B2

公开(公告)日：2023-04-18

申请号：US16868237

申请日：2020-05-06

申请人： UNIVERSITY OF MASSACHUSETTS

发明人： Robert H. Brown, Jr. ,  Jonathan K. Watts ,  Helene Tran ,  Michael Moazami

IPC分类号： C12N15/113

摘要： The present disclosure provides antisense compounds, methods, and compositions for silencing C9ORF72 transcripts. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with C9ORF72 in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with C9ORF72.

公开(公告)号：US10954518B2

公开(公告)日：2021-03-23

申请号：US16295621

申请日：2019-03-07

申请人： University of Massachusetts

发明人： Christian Mueller ,  Robert H. Brown, Jr.

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

摘要： 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：US10280418B2

公开(公告)日：2019-05-07

申请号：US15126688

申请日：2015-03-18

申请人： University of Massachusetts

发明人： Christian Mueller ,  Robert H. Brown, Jr.

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

摘要： 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：US10258668B2

公开(公告)日：2019-04-16

申请号：US15023044

申请日：2014-03-12

申请人： THE GENERAL HOSPITAL CORPORATION ,  MASSACHUSETTS EYE AND EAR INFIRMARY ,  UNIVERSITY OF MASSACHUSETTS

发明人： Patricia K. Donahoe ,  Demetrios Vavvas ,  David Pepin ,  Mien Van Hoang ,  Robert H. Brown, Jr. ,  Guangping Gao

IPC分类号： A61K38/22 ,  C07K16/26 ,  C07K14/575 ,  A61K48/00 ,  C07K14/765 ,  C12N7/00 ,  C12N15/86

摘要： The present invention relates to methods to treat a neurodegenerative disease or disorder, e.g., a motor neuron disease in a subject, whereby the subject is administered a recombinant human Mullerian Inhibiting Substance (MIS) protein as disclosed herein, wherein the recombinant human MIS protein comprises a modified Kex cleavage site for increased cleavage. The recombinant human MIS protein can be produced from a pre-proprotein comprising a non-MIS leader sequence or a functional fragment thereof in place of the MIS leader sequence.

公开(公告)号：US20240011027A1

公开(公告)日：2024-01-11

申请号：US17914706

申请日：2021-03-25

申请人： President and Fellows of Harvard College ,  University of Massachusetts

发明人： Kevin C. Eggan ,  Joseph Robert Klim ,  Robert H. Brown, Jr. ,  Jonathan K. Watts

IPC分类号： C12N15/113 ,  A61P25/28

CPC分类号： C12N15/113 ,  A61P25/28 ,  C12N2310/11 ,  C12N2310/316 ,  C12N2310/3231 ,  C12N2320/33

摘要： The disclosure relates to compositions and methods for treating a disease or condition associated with a TDP-pathology or a decline in TDP-43 functionality in neuronal cells in a subject, and for identifying candidate agents to suppress or prevent inclusion of an abortive or altered STMN2 RNA sequence.

公开(公告)号：US11827886B2

公开(公告)日：2023-11-28

申请号：US16872448

申请日：2020-05-12

申请人： University of Massachusetts

发明人： Robert H. Brown, Jr. ,  Janice A. Dominov

IPC分类号： C12N15/113 ,  C12Q1/6883 ,  C07K16/18 ,  C12N15/86 ,  G01N33/68

CPC分类号： C12N15/1138 ,  C07K16/18 ,  C12N15/86 ,  C12Q1/6883 ,  G01N33/6872 ,  C07K2317/34 ,  C12N2310/11 ,  C12N2320/33 ,  C12N2320/53 ,  C12N2750/14143 ,  C12Q2600/156 ,  G01N2333/705 ,  G01N2800/2878

摘要： Aspects of the disclosure relate to methods of altering RNA splicing in a subject. In some embodiments, methods are provided for correcting splicing in a cell that contains a DYSF gene having a mutation that results in defective splicing.

公开(公告)号：US11760999B2

公开(公告)日：2023-09-19

申请号：US17174452

申请日：2021-02-12

申请人： University of Massachusetts

发明人： Christian Mueller ,  Robert H. Brown, Jr.

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

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

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