公开(公告)号：US20230002787A1

公开(公告)日：2023-01-05

申请号：US17847291

申请日：2022-06-23

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves

IPC: C12N15/86 ,  C07K14/47 ,  A61P35/00

Abstract: Aspects of the disclosure relate to compositions and methods of treating certain genetic disease (e.g., Neurofibromatosis type I) by delivering functional neurofibromin 1(NF1) protein (e.g., mini-NF1 protein and/or full-length NF1 protein) to target cell (e.g., cells and/or tissue of a subject). The disclosure is based, in part, on isolated nucleic acids (e.g., rAAV vectors) and rAAVs engineered to express a functional NF1 protein (e.g., mini-NF1 protein and/or full-length NF1 protein) or variants thereof.

公开(公告)号：US20210238233A1

公开(公告)日：2021-08-05

申请号：US17050475

申请日：2019-04-26

Applicant: University of Massachusetts ,  The Johns Hopkins University ,  Kennedy Krieger Institute, Inc.

Inventor： Miguel Sena Esteves ,  Sourav Roy Choudhury ,  Kathryn Rae Wagner ,  Jennifer Gifford Green ,  Ana Rita Batista

IPC: C07K14/01 ,  C12N7/00

Abstract: The disclosure relates, in some aspects, to adeno-associated vims capsid proteins isolated from an in vivo library and recombinant adeno-associated viruses (rAAVs) comprising the same. In some aspects, the disclosure relates to isolated nucleic acids encoding AAV capsid proteins isolated from an in vivo library. In some embodiments, rAAVs and compositions described by the disclosure are useful for delivery of one or more transgenes to the muscle-tissue of a subject.

公开(公告)号：US20210095314A1

公开(公告)日：2021-04-01

申请号：US17046756

申请日：2019-04-12

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Diane Golebiowski

IPC: C12N15/86 ,  C12N9/24 ,  A61P25/00

Abstract: Aspects of the disclosure relate to bicistronic AAV nucleic acid constructs comprising a transgene encoding hexosaminidase A (HEXA) and hexosaminidase (HERB) proteins. In some embodiments, the disclosure provides methods for treating or preventing lysosomal storage disorders, such as Tay-Sachs disease and Sandhoff disease, using bicistronic nucleic acid constructs described by the disclosure.

公开(公告)号：US20190389934A1

公开(公告)日：2019-12-26

申请号：US16451049

申请日：2019-06-25

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Sourav Roy Choudhury

IPC: C07K14/775 ,  C12N15/79 ,  C07K19/00 ,  C07K14/075 ,  C07K7/04 ,  A61K48/00 ,  C12N15/86 ,  C07K14/005 ,  C07K14/00 ,  C07K7/00

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US20190038773A1

公开(公告)日：2019-02-07

申请号：US16074968

申请日：2017-02-02

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Ana Rita Batista ,  Sourav Roy Choudhury

IPC: A61K48/00 ,  A61K47/42

Abstract: The disclosure relates, in some aspects, to compositions and methods for enhanced delivery of a transgene to the central nervous system (CNS) of a subject. In some embodiments, the transgene is delivered by recombinant AAV (rAAV). In some embodiments, the method of enhancing transgene delivery comprises administering a blood brain barrier (BBB)-crossing molecule (e.g., K16ApoE) and an rAAV comprising a transgene to a subject.