公开(公告)号：US11014976B2

公开(公告)日：2021-05-25

申请号：US16451049

申请日：2019-06-25

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Sourav Roy Choudhury

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

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：US10370432B2

公开(公告)日：2019-08-06

申请号：US15516582

申请日：2015-10-02

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Sourav Roy Choudhury

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

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：US20250135033A1

公开(公告)日：2025-05-01

申请号：US18834366

申请日：2023-01-31

Applicant: University of Massachusetts

Inventor： Heather Gray-Edwards ,  Miguel Sena Esteves ,  Jillian Gallagher

IPC: A61K48/00 ,  A61K38/47 ,  A61K38/48 ,  A61P3/00 ,  C12N15/86

Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional NEU1 protein in a subject. In other aspects, the disclosure provides compositions and methods for treating sialidosis, galactosialidosis, and/or Alzheimer's Disease in a subject having or suspected of having sialidosis, galactosialidosis, and/or Alzheimer's Disease.

公开(公告)号：US11680275B2

公开(公告)日：2023-06-20

申请号：US16619733

申请日：2018-06-06

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Miguel Sena Esteves ,  Guangping Gao ,  Michael R. Green ,  Dan Wang ,  Tessa Mercedes Simone

IPC: C12N15/86 ,  C07K14/47

CPC classification number: C12N15/86 ,  C07K14/4702 ,  C07K2319/41 ,  C12N2310/141 ,  C12N2330/10 ,  C12N2750/14143

Abstract: In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.

公开(公告)号：US20220185862A1

公开(公告)日：2022-06-16

申请号：US17433269

申请日：2020-02-24

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Scot A. Wolfe

IPC: C07K14/705 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

Abstract: In some aspects, the disclosure relates to recombinant adeno-associated viruses (rAAVs) comprising a nucleic acid encoding a fusion protein comprising a DNA-binding domain and a transcriptional regulator domain and methods of using the same. In some embodiments, expression of the fusion protein results in modified expression of a target gene in a cell.

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.

公开(公告)号：US10711270B2

公开(公告)日：2020-07-14

申请号：US15516585

申请日：2015-10-02

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Sourav Roy Choudhury

IPC: C12N15/10 ,  C07K14/005 ,  C40B40/08

Abstract: Aspects of the disclosure relate to barcoded chimeric adeno-associated virus (AAV) capsid libraries, chimeric capsids and related recombinant AAVs (rAAVs) identified using the libraries. Specifically, the chimeric AAV capsid libraries comprise a plurality of nucleic adds encoding AAV capsid proteins, wherein each nucleic acid (i) encodes a unique AAV capsid protein having distinct polypeptide regions of greater than six amino acids in length that are derived from at least two different AAV serotypes, and (ii) comprises a unique barcode sequence. Further disclosed are methods of preparing an AAV library and identifying AAV capsids tropic for a target tissue.

公开(公告)号：US12291719B2

公开(公告)日：2025-05-06

申请号：US17237095

申请日：2021-04-22

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Sourav Roy Choudhury

IPC: C12N15/864 ,  A61K48/00 ,  C07K7/00 ,  C07K7/04 ,  C07K14/005 ,  C07K14/775 ,  C07K16/00 ,  C12N15/79 ,  C12N15/85 ,  C12N15/86 ,  C07K14/00 ,  C07K14/075 ,  C07K19/00

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：US20230416779A1

公开(公告)日：2023-12-28

申请号：US18311367

申请日：2023-05-03

Applicant: University of Massachusetts

Inventor： Miguel Sena Esteves ,  Guangping Gao ,  Michael R. Green ,  Dan Wang ,  Tessa Mercedes Simone

IPC: C12N15/86 ,  C07K14/47

CPC classification number: C12N15/86 ,  C07K14/4702 ,  C12N2750/14143 ,  C12N2310/141 ,  C12N2330/10 ,  C07K2319/41

Abstract: In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.

公开(公告)号：US11826433B2

公开(公告)日：2023-11-28

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 ,  A61K31/7016 ,  A61K31/351 ,  A61K47/64 ,  C12N15/79 ,  C12N15/86 ,  C07K14/775 ,  A61K47/62 ,  A61K31/5415 ,  C12N15/63 ,  A61K38/17 ,  A61K9/00 ,  C12N15/64 ,  A61K31/4706 ,  A61K47/42 ,  C07K5/083

CPC classification number: A61K48/0008 ,  A61K9/0085 ,  A61K31/351 ,  A61K31/4706 ,  A61K31/5415 ,  A61K31/7016 ,  A61K38/1709 ,  A61K47/42 ,  A61K47/62 ,  A61K47/6455 ,  C07K14/775 ,  C12N15/63 ,  C12N15/64 ,  C12N15/79 ,  C12N15/86 ,  C07K5/0808 ,  C07K2319/00 ,  C07K2319/33 ,  C12N2750/14143

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.