Suicide gene therapy: possible applications in haematopoietic disorders

Gene therapy for inherited disorders of haematopoietic cells.

Over the last decade, the majority of inborn errors of haematopoiesis has been elucidated on a molecular level. For some of these diseases, gene transfer into transplantable cells offers new therapeutic perspectives. Improved retroviral or lentiviral techniques allow stable gene transfer in >10% of repopulating cells cultured in vitro. However, severe impediments are still encountered with resp...

Suicide Gene Therapy against Cancer

A major limitation of conventional chemotherapies used in cancer treatments today are low therapeutic indices and side effects that result from drug effects on normal tissues (off target). One of the most innovative approaches to developing antineoplastic agents with increased tumor selectivity is the use of suicide gene therapy. Suicide gene therapy involves delivering a gene product in proxim...

Purification and characterization of Aspergillus parasiticus cytosine deaminase for possible deployment in suicide gene therapy

Cytosine Deaminase (CD) from Aspergillus parasiticus was purified and characterized. Time course for maximal CD production (50 μmol/min/mg) was at 72 hrs. The enzyme was purified 387.73 folds with an overall yield of 13%. The CD had pH optimum of 7.2, a temperature optimum of 40 ̊C 45 ̊C, activation energy (Ea) of 8.4 KJ/mol and a t1/2 of 1.10 hr. A. parasiticus CD stoichiometrically deaminated C...

Prodrug-activating systems in suicide gene therapy.

Use of suicide genes in gene therapy.

Gene therapy encompasses a broad range of treatment modalities that may eventually be applied to a variety of genetic as well as acquired diseases. In addition to compensating for a defective gene--and enhancing a cellular function--gene transfer can allow for a conditional negative selection of target cells. Indeed, the transfer of a gene encoding a susceptibility factor can make a cell specif...