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Gene Therapist


Gene therapy experts use genes or genetic material as curative elements in order to correct or alter the function of genes that malfunction at the most basic level, DNA. You could say that they use genes instead of medication, and introduce them into the patient's cells in order to achieve therapeutic effects. Gene therapy is a new medical treatment which is emerging on the back of new knowledge which has been developed in the fields of genomics, genetic engineering and other areas of biotechnologies and bioinformatics. At present, gene therapy experts carry out their work at the research stage, for both public and private centres, investigating possible diseases that should be treated with this type of therapy and fine-tuning the safest and most effective techniques for the introduction or suppression of the target genes. Diseases that could be treated with these types of therapy are those with a genetic basis (whether congenital or acquired), many of which are untreatable today, including several varieties of cancer, cystic fibrosis and many neurological diseases.


  • Analyse genetic material, identifying wich fragments of DNA make up each gene, possible mutations of these, and which genes are involved in the development of certain diseases.
  • Once the genes involved in a disease have been identified, study and design different procedures in order to successfully replace the genetic mutations responsible. The success of these procedures depends to a large extent on the choice and development of the most suitable type of vector in order to transfer the therapeutic genes. It must be possible to produce large quantities of the vector, which should be able to reach the deliver DNA intact to the nucleus of the cell needing treatment. The vectors which deliver the genes can be viral or synthetic. In the case of viral vectors, the pathogenic function must be eliminated.
  • Design (or take part in the design) and oversee (or carry out) preclinical trials (in vitro and on animals) to test the production of the vector, the effectiveness of introducing the gene and the lack of side effects and/or adverse reactions. Notify and obtain approval from the relevant Committee on Animal Research and Ethics.
  • Once satisfactory results have been obtained from multiple preclinical trials, and all of the necessary licences have been obtained from the various regulatory authorities, gene therapy experts devise and schedule clinical trials (at this point on human subjects) and conduct or oversee these. The first clinical trials aim above all to check that there is good tolerance of the vector that carries the gene. The planning, conducting, records and communication of clinical trials must all be carried out in line with the internationally recognised ethical and scientific quality standards known as Good Clinical Practice, and with the approval of the Clinical Research Ethical Committee. In this phase they work extremely closely with the clinical coordinator and/or clinical trial monitor.
  • Shortly afterwards, they will be responsible for designing a specific treatment of each patient affected by genetic diseases.
  • These professionals must continuously update and share their constantly developing knowledge in the fields of genomics and biotechnologies. Gene therapy experts regularly attend and take part in international congresses.

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Generalitat de Catalunya
Unió Europea FEDER
Unió Europea FSE