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    Gene expression control DNA element and associated protein
    6.
    发明授权
    Gene expression control DNA element and associated protein 有权
    基因表达控制DNA元件和相关蛋白

    公开(公告)号：US07371540B2

    公开(公告)日：2008-05-13

    申请号：US09966264

    申请日：2001-09-28

    Applicant: Elizabeth K. Barber

    Inventor： Elizabeth K. Barber

    IPC: C12P21/06 ,  C12N5/00 ,  C12N15/00 ,  C07H21/02 ,  C07H21/04

    CPC classification number: C07K14/705 ,  A61K48/00 ,  C07K14/4708 ,  C07K16/3061 ,  G01N33/6887

    Abstract: Through screening of an expression library, a cDNA sequence has been identified that encodes a protein that interacts with human CD33, the DNA being highly homologous to a portion of the human dystrophin gene. A region of that cDNA has been identified as an important regulatory element in controlling expression, both transcription and translation, of the DNA with which it is associated. This DNA sequence element may be used as a regulatory cassette in conjunction with any suitable gene, to modify gene expression. The putative controlling DNA sequence element contains a minimum of 137 base pairs (FIG. 1) to 147 base pairs (FIG. 1A) and a maximum of 287 base pairs (FIG. 1B).

    Abstract translation: 通过筛选表达文库，已经鉴定了编码与人CD33相互作用的蛋白质的cDNA序列，该DNA与人肌营养不良蛋白基因的一部分高度同源。 该cDNA的区域已被确定为控制与其相关的DNA的转录和翻译的重要调控元件。 该DNA序列元件可以与任何合适的基因一起用作调节盒，以修饰基因表达。 推定的控制DNA序列元件包含至少137个碱基对（图1）至147个碱基对（图1A）和最多287个碱基对（图1B）。

    Mini -dystrophin nucleic acid and peptide sequences
    7.
    发明申请
    Mini -dystrophin nucleic acid and peptide sequences 有权
    迷你抗肌营养不良蛋白核酸和肽序列

    公开(公告)号：US20030216332A1

    公开(公告)日：2003-11-20

    申请号：US10149736

    申请日：2002-11-25

    Inventor： Jeffrey S Chamberlain ,  Scott Q Harper

    IPC: A61K048/00 ,  C12N015/85

    CPC classification number: C07K14/4708 ,  A01K2217/05 ,  A61K48/00 ,  C12N2799/022

    Abstract: The present invention relates to compositions and methods for expressing mini-dystrophin peptides. In particular, the present invention provides compositions comprising nucleic acid sequences that are shorter than wild-type dystrophin cDNA and that express mini-dystrophin peptides that function in a similar manner as wild-type dystrophin proteins. The present invention also provides compositions comprising mini-dystrophin peptides, and methods for expressing mini-dystrophin peptides in target cells.

    Abstract translation: 本发明涉及用于表达微型肌营养不良蛋白肽的组合物和方法。 特别地，本发明提供了包含比野生型肌营养不良蛋白cDNA短的核酸序列并且表达与野生型肌营养不良蛋白类似的功能的微型肌营养不良蛋白肽的组合物。 本发明还提供了包含迷你抗肌营养不良蛋白肽的组合物，以及用于在靶细胞中表达微型肌营养不良蛋白肽的方法。

    DNA sequences encoding dystrophin minigenes and methods of use thereof
    8.
    发明申请
    DNA sequences encoding dystrophin minigenes and methods of use thereof 有权

    公开(公告)号：US20030171312A1

    公开(公告)日：2003-09-11

    申请号：US09845416

    申请日：2001-04-30

    Inventor： Xiao Xiao

    IPC: A61K048/00 ,  C07H021/04

    CPC classification number: C07K14/4708 ,  A61K38/00 ,  C12N2799/025

    Abstract: The present invention provides a series of novel dystrophin minigenes that retain the essential biological functions. The expression of the dystrophin minigenes may be controlled by a regulatory element along with a small polyadenylation signal. The entire gene expression cassettes may be readily packaged into a viral vector, preferably an AAV vector. The present invention further defines the minimal functional domains of dystrophin and provides ways to optimize and create new versions of dystrophin minigenes. Finally, the present invention provides a method of treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

    Gene expression control DNA element and associated protein
    9.
    发明申请
    Gene expression control DNA element and associated protein 有权
    基因表达控制DNA元件和相关蛋白

    公开(公告)号：US20020099015A1

    公开(公告)日：2002-07-25

    申请号：US09966264

    申请日：2001-09-28

    Inventor： Elizabeth K. Barber

    IPC: A61K038/17 ,  C07H021/04 ,  C07K014/435 ,  C12P021/02 ,  C12N005/06

    CPC classification number: C07K14/705 ,  A61K48/00 ,  C07K14/4708 ,  C07K16/3061 ,  G01N33/6887

    Abstract: Through screening of an expression library, a cDNA sequence has been identified that encodes a protein that interacts with human CD33, the DNA being highly homologous to a portion of the human dystrophin gene. A region of that cDNA has been identified as an important regulatory element in controlling expression, both transcription and translation, of the DNA with which it is associated. This DNA sequence element may be used as a regulatory cassette in conjunction with any suitable gene, to modify gene expression. The putative controlling DNA sequence element contains a minimum of 137 base pairs (FIG. 1) to 147 base pairs (FIG. 1A) and a maximum of 287 base pairs (FIG. 1B).

    Abstract translation: 通过筛选表达文库，已经鉴定了编码与人CD33相互作用的蛋白质的cDNA序列，该DNA与人肌营养不良蛋白基因的一部分高度同源。 该cDNA的区域已被确定为控制与其相关的DNA的转录和翻译的重要调控元件。 该DNA序列元件可以与任何合适的基因一起用作调节盒，以修饰基因表达。 推定的控制DNA序列元件包含至少137个碱基对（图1）至147个碱基对（图1A）和最多287个碱基对（图1B）。

    Adenovirus-medicated gene transfer to cardiac and vascular smooth muscle
    10.
    发明授权
    Adenovirus-medicated gene transfer to cardiac and vascular smooth muscle 失效
    腺病毒药物基因转移到心脏和血管平滑肌

    公开(公告)号：US06297220B1

    公开(公告)日：2001-10-02

    申请号：US08972831

    申请日：1997-11-18

    Applicant: Jeffrey M. Leiden ,  Eliav Barr

    Inventor： Jeffrey M. Leiden ,  Eliav Barr

    IPC: C12N700

    CPC classification number: C12N15/86 ,  A61K48/00 ,  C07K14/4708 ,  C12N2710/10343 ,  C12N2830/008

    Abstract: The present invention relates to the use of adenovirus-mediated gene transfer to regulate function in cardiac and vascular smooth muscle cells. A recombinant adenovirus comprising a DNA sequence that codes for a gene product is delivered to a cardiac or vascular smooth muscle cell and the cell is maintained until that gene product is expressed. Delivery is direct injection into a muscle cell or infusing a pharmaceutical composition containing an adenovirus virus vector construct intravascularly.

    Abstract translation: 本发明涉及腺病毒介导的基因转移在心脏和血管平滑肌细胞中调节功能的用途。 将包含编码基因产物的DNA序列的重组腺病毒递送至心脏或血管平滑肌细胞，并维持细胞，直至该基因产物被表达。 输送是直接注射到肌肉细胞中或灌注含有血管内的腺病毒载体构建体的药物组合物。

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