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Type: Journal article
Title: Refinement of lentiviral vector for improved RNA processing and reduced rates of self inactivation repair
Author: Koldej, R.
Anson, D.
Citation: BMC Biotechnology, 2009; 9(86):1-13
Publisher: BioMed Central Ltd.
Issue Date: 2009
ISSN: 1472-6750
Statement of
Rachel M Koldej and Donald S Anson
Abstract: Background Lentiviral gene therapy vectors are now finding clinical application. In order to fully exploit their potential it is important that vectors are made as efficient and as safe as possible. Accordingly, we have modified a previously reported vector to improve RNA processing, minimise Human Immunodeficiency Virus Type-1 (HIV-1) sequence content and reduce repair of the self inactivating (SIN) deletion. Results HIV-1 sequence in the vector was reduced by substituting the polyadenylation signal with a heterologous signal. Mutation of splice donor sites was undertaken to prevent the majority of splicing within the vector genomic RNA. In addition, a number of other sequences within the vector were deleted. The combination of these modifications was able to significantly reduce the rates of both vector mobilisation and repair of the self inactivating deletion after two rounds of marker rescue. Conclusion RNA processing can be improved by mutation of the major and minor HIV-1 splice donor sites in the vector. In addition the rate of vector mobilisation and repair of SIN vectors can be successfully reduced by careful vector design that reduces homology between the 5' and 3' long terminal repeats (LTRs) to a minimum.
Keywords: Cell Line
RNA Splice Sites
RNA, Viral
Genetic Vectors
Genetic Therapy
DOI: 10.1186/1472-6750-9-86
Appears in Collections:Aurora harvest
Paediatrics publications

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