Rikki Solow

US Patent:

61657816, Dec 26, 2000

Filed:

Jan 21, 1999

Appl. No.:

9/235375

Inventors:

Barrie J. Carter - Seattle WA
Terence Flotte - Gainesville FL
Sandra Afione - Rockville MD
Rikki Solow - Gaithersburg MD

Assignee:

The United States of America as represented by the Department of Health
and Human Services - Washington DC

International Classification:

C12N 15864
C07H 2104

US Classification:

4353201

Abstract:

Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

US Patent:

58666966, Feb 2, 1999

Filed:

Apr 3, 1996

Appl. No.:

8/626953

Inventors:

Barrie J. Carter - Kensington MD
Terence Flotte - Baltimore MD
Sandra Afione - Bethesda MD
Rikki Solow - Gaithersburg MD

Assignee:

The United States of America as represented by the Department of Health
and Human Services - Washington DC

International Classification:

C07H 2104

US Classification:

536 235

Abstract:

Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

US Patent:

59902795, Nov 23, 1999

Filed:

May 31, 1995

Appl. No.:

8/455552

Inventors:

Barrie J. Carter - Kensington MD
Terence Flotte - Baltimore MD
Sandra Afione - Bethesda MD
Rikki Solow - Gaithersburg MD

Assignee:

The United States of America as represented by the Department of Health
and Human Services - Washington DC

International Classification:

C07K 1400
C12P 2106

US Classification:

530350

Abstract:

Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

US Patent:

55873081, Dec 24, 1996

Filed:

Jun 2, 1992

Appl. No.:

7/891962

Inventors:

Barrie J. Carter - Kensington MD
Terence Flotte - Baltimore MD
Sandra Afione - Bethesda MD
Rikki Solow - Gaithersburg MD

Assignee:

The United States of America as represented by the Department of Health
& Human Services - Washington DC

International Classification:

C12N 510
C12N 1586
C07H 2104

US Classification:

4352402

Abstract:

Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

US Patent:

56587767, Aug 19, 1997

Filed:

Jun 7, 1995

Appl. No.:

8/448613

Inventors:

Terence R. Flotte - Glen Burnie MD
Barrie J. Carter - Seattle WA
William B. Guggino - Baltimore MD
Rikki Solow - Gaithersburg MD

Assignee:

Targeted Genetics Corporation - Seattle WA
Johns Hopkins University - Baltimore MD

International Classification:

C12N 516
C12N 1564
C12N 1586

US Classification:

4351723

Abstract:

Adeno-associated virus (AAV) vectors may have utility for gene therapy but heretofore a significant obstacle has been the inability to generate sufficient quantifies of such recombinant vectors in amounts that would be clinically useful for human gene therapy application. Stable, helper-free AAV packaging cell lines have been elusive, mainly due to the activities of Rep protein, which down-regulates its own expression and reverses cellular immortalization. This invention provides packaging systems and processes for packaging AAV vectors that efficiently circumvent these problems by replacing the AAV p5 promoter with a heterologous promoter and that allow for substantially increased packaging efficiency.

US Patent:

59895403, Nov 23, 1999

Filed:

May 31, 1995

Appl. No.:

8/455231

Inventors:

Barrie J. Carter - Kensington MD
Terence Flotte - Baltimore MD
Sandra Afione - Bethesda MD
Rikki Solow - Gaithersburg MD

Assignee:

The United States of America as represented by the Department of Health
& Human Services - Washington DC

International Classification:

A61K 4800
C12N 1564
C12N 1586

US Classification:

424 932

Abstract:

Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.