公开(公告)号：US20250025575A1

公开(公告)日：2025-01-23

申请号：US18712552

申请日：2022-11-23

Applicant: THE TRUSTEES OF PRINCETON UNIVERSITY

Inventor： Carola Maturana ,  Esteban Engel

IPC: A61K48/00 ,  C12N15/86

Abstract: Provided are polynucleotides comprising a transcriptional control element comprising a promoter sequence having at least 80% sequence identity to SEQ ID NO: 1. SEQ ID NO:2. SEQ ID NO:3 or SEQ ID NO:4 and a cell- or tissue-specific enhancer element. Also provided are compositions (e.g., vectors, viruses, host cells, and pharmaceutical compositions) comprising said polynucleotides, methods that are useful for expressing a target gene in a host cell, and methods that are useful for expressing a target gene in a subject in need thereof.

公开(公告)号：US20220378941A1

公开(公告)日：2022-12-01

申请号：US17426389

申请日：2020-02-05

Applicant: The Trustees of Princeton University

Inventor： Esteban Engel ,  Carola Maturana ,  Lynn Enquist

IPC: A61K48/00 ,  C12N15/86 ,  C07K14/005

Abstract: Provided are recombinant nucleic acids (e.g., vectors), and related methods, for expression of a target gene in a host cell. The recombinant nucleic acids comprise a promoter comprising a nucleic acid sequence having at least 70% sequence identity to a sequence selected from SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6, SEQ ID NO: 7, SEQ ID NO: 8 and SEQ ID NO: 9; a cloning site for insertion of a nucleic acid encoding the target gene; and at least one non-promoter regulatory element required for the expression of the target gene in the host cell.