Chiron Corporation

Other

University of Iowa Research Foundation

Sybille L Sauter

Mordechai Bodner

Nicholas J DePolo

Beverly L Davidson

Thomas W Dubensky, Jr

Douglas J Jolly

David A Driver

James G Respess

Sunil D Chada

Colleen Stein

Jason A Heth

Stephen M W Chang

Philip Lee Sheridan

Gene delivery vectors, such as, for example, recombinant FIV vectors, and methods of using such vectors are provided for use in treating or preventing retinal diseases of the eye and diseases of the brain associated with lysosomal storage disorders.

Use of recombinant gene delivery vectors for treating or preventing lysosomal storage disorders

Gene delivery vectors, for example, recombinant FIV vectors, and methods of using such vectors are provided for use in treating or preventing retinal diseases of the eye and diseases of the brain associated with lysosomal storage disorders.

Retroviral vector particle producing cells are provided, wherein the cell (a) has greater than 5 stably integrated copies of a retroviral vector construct; (b) produces greater than 10 infectious recombinant retroviral vector particles per cell per day; and (c) produces replication incompetent retroviral vector particles.

Methods for producing a vector producing cell line utilizing a high multiplicity of transduction

Retroviral vector constructs are described which have a 5&prime; LTR, a tRNA binding site, a packaging signal, one or more heterologous sequences, an origin of second strand synthesis and a 3&prime; LTR, wherein the vector construct lacks retroviral gag/pol or env coding sequences. In addition, gag/pol, and env expression cassettes are described wherein the expression cassettes lack a consecutive sequence of more than 8 nucleotides in common. The above-described retroviral vector constructs, gag/pol and env expression cassettes may be utilized to construct producer cell lines which preclude the formation of replication competent virus.

Crossless retroviral vectors

Retroviral vector constructs are described which have a 5' LTR, a tRNA binding site, a packaging signal, one or more heterologous sequences, an origin of second strand synthesis and a 3' LTR, wherein the vector construct lacks retroviral gag/pol or env coding sequences. In addition, gag/pol, and env expression-cassettes are described wherein the expression cassettes lack a consecutive sequence of more than 8 nucleotides in common. The above-described retroviral vector constructs, gag/pol and env expression cassettes may be utilized to construct producer cell lines which preclude the formation of replication competent virus.

Growing community of inventors

Del Mar, CA, United States of America

Sybille L Sauter