The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Jun. 20, 2000

Filed:

May. 14, 1997
Applicant:
Inventors:

Lingxun Duan, North Wales, PA (US);

Mark A Zern, Newtown, PA (US);

Roger J Pomerantz, Chalfont, PA (US);

Assignee:

Thomas Jefferson University, Philadelphia, PA (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C07H / ; C12N / ; C12Q / ;
U.S. Cl.
CPC ...
4353201 ; 435-6 ; 435 9331 ; 536 231 ; 536 241 ; 536 245 ;
Abstract

Methods of gene therapy, particular ribozyme-mediated gene replacement methods, are disclosed. Method of treating patients suffering from a disease associated with expression of an abnormal form of a gene, such as alpha-1 antitrypsin mutations, are disclosed. The methods comprise the steps of administering to such a patient a nucleic acid construct encoding a ribozyme and a nucleic acid construct comprising a ribozyme resistant gene encoding a wild type form of the gene product. Recombinant vectors and pharmaceutical compositions for practicing the methods are disclosed.


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