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P M Lehn

Publications and source records attributed to P M Lehn.

3 recordsLinked to original sources
PubMed1990

Gene therapy using bone marrow transplantation: a 1990 update.

The use of recombinant retroviruses for gene transfer into the haematopoietic tissue in vivo is still a new area of research. The initial results of in vitro studies were very exciting. In contrast, the early in vivo studies in mice were somewhat disappointing because of the transient and low levels of expression of the transferred gene. Recently, however, better results have been obtained in the murine system in vivo. New packaging cell lines have been constructed, which are safer and still efficient. Better vectors have been designed. Thus, significant levels of expression of the transgene have been achieved in murine long-term transplant recipients. However, the results obtained so far in large animal studies are still disappointing. It should be emphasized that further progress must be based on a simple overall strategy involving better understanding of the functioning of the gene to be transferred by gene expression studies, design of vectors carrying a fully active and correctly regulated minilocus and better knowledge of the biological properties of the target cells, the haematopoietic stem cells.

Animals↗
PubMed1987

Gene therapy using bone marrow transplantation.

As a result of new techniques in molecular biology, the basic defects in a variety of genetic diseases have been characterized and the corresponding normal genes cloned. Methods have been devised for transferring specific genes with high efficiency into various cell types that exploit the use of replication-defective retroviruses as vectors. Bone marrow may be the ideal target for these attempts to transfer genes, because it contains self-renewing stem cells and because it can readily be collected by standard techniques, manipulated ex vivo as required with comparative ease and then reinfused. In a murine system, recent studies have shown that genes can indeed be incorporated into haemopoietic stem cells that can then completely reconstitute the marrows of syngeneic recipients. Thus far, such genes after transfer have not been appropriately expressed in vivo, although appreciable expression has been documented in cell culture studies in vitro. It is reasonable to expect that improvements in the design of retroviral vectors should permit appropriate expression of a transferred gene. When this is achieved, the use of bone marrow transplantation for gene transfer will offer new options for therapy.

Animals↗