公开(公告)号：US20210393714A1

公开(公告)日：2021-12-23

申请号：US17333304

申请日：2021-05-28

Applicant: University of Massachusetts

Inventor： Miguel Sena-Esteves ,  Cara Weismann ,  Diane Golebiowski ,  Lorelei Stoica

IPC: A61K35/761 ,  A61K48/00 ,  C12N15/86 ,  C12N9/24 ,  A61P25/28 ,  A61P43/00 ,  C12N9/38 ,  C12N15/861

Abstract: Aspects of the invention relate to recombinant adeno-associated viruses (rAAVs). In some aspects, the rAAVs comprise artificial genetic regulatory elements that modulate transgene expression. In some aspects, the disclosure relates to the treatment of lysosomal storage disorders.

公开(公告)号：US20180311290A1

公开(公告)日：2018-11-01

申请号：US15567847

申请日：2016-04-20

Applicant: University of Massachusetts

Inventor： Miguel Sena-Esteves ,  Cara Weismann ,  Diane Golebiowski ,  Lorelei Stoica

IPC: A61K35/761 ,  C12N9/38 ,  A61K48/00 ,  A61P25/28 ,  C12N15/861 ,  A61P43/00

CPC classification number: C12N15/86 ,  A61K35/761 ,  A61K48/00 ,  C12N9/2402 ,  C12N2750/14143

Abstract: Aspects of the invention relate to recombinant adeno-associated viruses (rAAVs). In some aspects, the rAAVs comprise artificial genetic regulatory elements that modulate transgene expression. In some aspects, the disclosure relates to the treatment of lysosomal storage disorders.

公开(公告)号：US11020443B2

公开(公告)日：2021-06-01

申请号：US15567847

申请日：2016-04-20

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Cara Weismann ,  Diane Golebiowski ,  Lorelei Stoica

IPC: A61K35/761 ,  A61K48/00 ,  A61P25/28 ,  A61P43/00 ,  C12N15/861 ,  C12N15/86 ,  C12N9/24 ,  C12N9/38

Abstract: Aspects of the invention relate to recombinant adeno-associated viruses (rAAVs). In some aspects, the rAAVs comprise artificial genetic regulatory elements that modulate transgene expression. In some aspects, the disclosure relates to the treatment of lysosomal storage disorders.