Development of globin expression vectors for human gene therapy

Objective

The long term correction of sickle cell anaemia and thalassaemia by gene therapy requires the stable integration of a fully functional a- or beta-globin gene into the genome of a sufficient number of bone marrow (stem) cells of the patient. To achieve this two major problems will have to be addressed, namely stable delivery and expression. At present the efficient introduction and stable integration of genes into a host cell genome can be achieved with known retroviral and adeno associated viral (AAV) vectors. The most efficient elements to express a gene in a stable manner are LCR's. The objective of this proposal is to construct a LCR containing beta-globin transcription unit and viral vector technology that could lead to gene therapy clinical trials. As an attractive alternative we will also start the development of a non viral gene transfer system based on ligand mediated gene transfer. This system has the potential advantage that it has less size constraints and that it can be produced on large scale.

Fields of science

• medical and health sciences › medical biotechnology › genetic engineering › gene therapy
• natural sciences › biological sciences › genetics › genomes

Programme(s)

• FP4-BIOMED 2 - Specific research, technological development and demonstration programme in the field of biomedicine and health, 1994-1998

Topic(s)

• 5.4 - Somatic gene therapy

Call for proposal

Data not available

Funding Scheme

CSC - Cost-sharing contracts

Coordinator

Erasmus Universiteit Rotterdam

Address

50,dr. Molewaterplein
3000 DR Rotterdam
Netherlands

Participants (4)

National and Kapodestrian University of Athens

Greece

Address

17,aghiou Thoma Street
11527 Athens

Therexsys Ltd

United Kingdom

Address

ST5 5SP Keele

United Medical and Dental Schools of Guy's and St Thomas's Hospitals

United Kingdom

Address

London Bridge
SE1 9RT London

Universita degli Studi di Cagliari

Italy

Address

Via Jenner S/n
09100 Cagliari