Abstract: A method of determining the safety and/or efficacy of a gene therapy vector prior to carrying out gene therapy on an individual includes infecting a culture of induced pluripotent stem cells obtained from the individual with a gene therapy vector. Ideally the cells are allowed to differentiate, and the infection is carried out at the start of culture and at least once during differentiation and/or after differentiation. Nucleic acids are extracted from the infected cells, and then analysed to measure and/or determine adverse effects of integration of nucleic acid from the vector and/or efficiency of expression of nucleic acid from the vector.

Abstract: The invention relates to the fabrication and use of silica organic nanoparticles as delivery vehicles for vims and virus-like species to the body. The nanoparticles typically have a hollow core and a surface morphology that allows effective adhesion of species to the surface for delivery to the body. In particular, the invention is particularly useful for performing transfection and transduction.

Abstract: A method of predicting the likelihood of success of a gene therapy procedure includes inducing DNA damage in a cell sample from an individual. The ability of the individual's cells in the sample to repair the DNA damage is then assessed to determine whether the individual could tolerate DNA damage caused by a gene therapy vector. In preferred embodiments, the ability of the individual's cells to repair DNA damage is assessed by detecting, and monitoring the subsequent disappearance of, a marker of DNA damage repair (such as gamma H2AX or phosphorylated 53BP1) in the sample.

Abstract: A method of predicting the likelihood of success of a gene therapy procedure includes inducing DNA damage in a cell sample from an individual. The ability of the individual's cells in the sample to repair the DNA damage is then assessed to determine whether the individual could tolerate DNA damage caused by a gene therapy vector. In preferred embodiments, the ability of the individual's cells to repair DNA damage is assessed by detecting, and monitoring the subsequent disappearance of, a marker of DNA damage repair (such as gamma H2AX or phosphorylated 53BP1) in the sample.

Abstract: A method of determining the safety and/or efficacy of a gene therapy vector prior to carrying out gene therapy on an individual includes infecting a culture of induced pluripotent stem cells obtained from the individual with a gene therapy vector. Ideally the cells are allowed to differentiate, and the infection is carried out at the start of culture and at least once during differentiation and/or after differentiation. Nucleic acids are extracted from the infected cells, and then analysed to measure and/or determine adverse effects of integration of nucleic acid from the vector and/or efficiency of expression of nucleic acid from the vector.

Abstract: A method of producing a transgenic cell comprising introducing into a cell a non-primate lentiviral expression vector comprising a nucleotide of interest (NOI). Also described is a method of producing a transgenic cell comprising introducing into a cell a lentiviral expression vector comprising a NOI capable of generating an antisense oligonucleotide, a ribozyme, an siRNA, a short hairpin RNA, a micro-RNA or a group 1 intron. Also described is a viral vector comprising a first nucleotide sequence, wherein said first nucleotide sequence comprises: (a) a second nucleotide sequence comprising an aptazyme; and (b) a third nucleotide sequence capable of generating a polynucleotide; wherein (a) and (b) are operably linked and wherein the aptazyme is activatable to cleave a transcript of the first nucleotide sequence such that said polynucleotide is generated.

Abstract: Combinations and methods for inducing a semi-synchronous wave of liver cell proliferation in vivo and combinations and methods for inducing a semi-synchronous wave of liver cell proliferation and achieving transduction of proliferating liver cells in vivo are disclosed.

Abstract: A method of producing a transgenic cell comprising introducing into a cell a non-primate lentiviral expression vector comprising a nucleotide of interest (NOI). Also described is a method of producing a transgenic cell comprising introducing into a cell a lentiviral expression vector comprising a NOI capable of generating an antisense oligonucleotide, a ribozyme, an siRNA, a short hairpin RNA, a micro-RNA or a group 1 intron. Also described is a viral vector comprising a first nucleotide sequence, wherein said first nucleotide sequence comprises: (a) a second nucleotide sequence comprising an aptazyme; and (b) a third nucleotide sequence capable of generating a polynucleotide; wherein (a) and (b) are operably linked and wherein the aptazyme is activatable to cleave a transcript of the first nucleotide sequence such that said polynucleotide is generated.

Abstract: A method of producing a transgenic cell comprising introducing into a cell a non-primate lentiviral expression vector comprising a nucleotide of interest (NOI).

Abstract: Combinations and methods for inducing a semi-synchronous wave of liver cell proliferation in vivo and combinations and methods for inducing a semi-synchronous wave of liver cell proliferation and achieving transduction of proliferating liver cells in vivo are disclosed.

Abstract: Combinations and methods for inducing a semi-synchronous wave of liver cell proliferation in vivo and combinations and methods for inducing a semi-synchronous wave of liver cell proliferation and achieving transduction of proliferating liver cells in vivo are disclosed.