Chiron Corporation

Nicholas J DePolo

James G Respess

Mordechai Bodner

Sybille L Sauter

David A Driver

Sunil D Chada

Douglas J Jolly

Stephen Chang

Carlos E Ibanez

Stephen M W Chang

David Chi-Tang Hsu

Philip Lee Sheridan

Lee Will

Judith Greengard

Methods are provided for obtaining measurable levels of a protein, nucleic acid molecule, or enzymatic product in a bodily fluid or cells of a human, comprising the step of administering to a human a recombinant retroviral preparation having a titer on HT1080 cells of greater than 10 cfu/ml, wherein the recombinant retroviral preparation is capable of directing the expression of a protein, nucleic acid molecule, or enzyme which generates an enzymatic product, such that measurable levels of the protein, nucleic acid molecule, or enzymatic product may be obtained in the bodily fluid or cells of the human.

Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other 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.

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Solano Beach, CA, United States of America

Nicholas J DePolo