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：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.