公开(公告)号：US20250011812A1

公开(公告)日：2025-01-09

申请号：US18712273

申请日：2022-11-22

Applicant: University of Massachusetts

Inventor： Jun Xie ,  Guangping Gao ,  Qing Xie ,  Hong Ma

IPC: C12N15/86 ,  A61K48/00 ,  A61P25/00

Abstract: Aspects of the disclosure relate to compositions and methods for treating spinal muscular atrophy (SMA). The disclosure is based, in part, on isolated nucleic acids and vectors (e.g., viral vectors, such as rAAV vectors) encoding SMN1. In some embodiments, the expression of SMN 1 is driven by a native SMN 1 promoter or a variant thereof. In some embodiments, isolated nucleic acids and vectors of the disclosure have reduced toxicity and/or increased transgene expression relative to previously described SMN-encoding vectors.

公开(公告)号：US20240424144A1

公开(公告)日：2024-12-26

申请号：US18827471

申请日：2024-09-06

Applicant: University of Massachusetts

Inventor： Jun Xie ,  Guangping Gao ,  Qing Xie ,  Hong Ma

IPC: A61K48/00 ,  A61P21/00 ,  C12N15/86

Abstract: Aspects of the disclosure relate to compositions and methods for treating spinal muscular atrophy (SMA). The disclosure is based, in part, on isolated nucleic acids and vectors (e.g., viral vectors, such as rAAV vectors) encoding SMN1. In some embodiments, the expression of SMN1 is driven by a native SMN1 promoter or a variant thereof. In some embodiments, isolated nucleic acids and vectors of the disclosure have reduced toxicity and/or increased transgene expression relative to previously described SMN-encoding vectors.