Johnson & Johnson Research Pty Limited

Gene Shears Pty. Limited

Lun-Quan Sun

Geoffrey Phillip Symonds

Wayne Lyle Gerlach

Rafael G Amado

Gregory C Fanning

Janet L MacPherson

Janet Lesley Macpherson

Greg Fanning

Geoffery P Symonds

Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invention particularly relates to ex vivo gene therapy of HP cells for treatment or prevention of HIV infection.

Process for the preparation of a composition of genetically modified hematopoietic progenitor cells

Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells

A cell comprising a synthetic non-naturally occurring oligonucleotide compound comprises nucleotides whose sequence defines a conserved catalytic region and nucleotides whose sequence hybridizes with a predetermined target sequence within a MoMLV Psi packaging sequence on the HIV tat sequence. The catalytic region may be derived from a hammerhead ribozyme, a hairpin ribozyme a hepatitis delta ribozyme, an PNAase P ribozyme, a group I intron or a group II intron.

Ribozymes targeting the MoMLV PSI packaging sequence and the HIV tat

This invention is directed to a synthetic non-naturally occurring oligonucleotide compound which comprises nucleotides whose sequence defines a conserved catalytic region and nucleotides whose sequence is capable of hybridizing with a predetermined target sequence within a packaging sequence of an RNA virus. Preferably, the viral packaging sequence is a retrovirus packaging sequence or the HIV-1 Psi packaging sequence. The RNA virus may be HIV-1, Feline Leukemia Virus, Feline Immunodeficiency Virus or one of the viruses listed in Table I. The conserved catalytic region may be derived from a hammerhead ribozyme, a hairpin ribozyme, a hepatitis delta ribozyme, an RNAase P ribozyme, a group I intron, a group II intron. The invention is also directed to multiple ribozymes, combinations of ribozymes, with or without antisense, and combinations of ribozymes, with antisense, and TAR decoys, poly TARs or RRE decoys targeted against the RNA virus and combinations of ribozymes and antisense targetted against the RNA virus. Vectors are also described. Further, methods of treatment and methods of use both in vivo and ex vivo are described.

Ribozymes targeting retroviral packaging sequence expression constructs

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Eastwood, Australia

Lun-Quan Sun