Targeting and Controlling Genes: The New Challenges for Cell and Gene Therapy

I n the last months, the first successful clinical trial based on lentivirus-mediated gene therapy of hematopoietic stem cells for the treatment of X-linked adrenoleukodystrophy, a severe demyelinating brain disease, has been reported. In parallel, reprogramming of somatic cells into induced pluripotent stem (iPS) cells, using integrating and nonintegrating lentivirus-derived polycistronic vectors, has also been reported. These encouraging advances allow the prediction of future preclinical and clinical developments for the treatment of metabolic and degenerative brain disorders, and also will enhance research in the field of iPS cells with the goal of developing new approaches for regenerative medicine. For such reasons, the board of the Société Francophone de Thérapie Cellulaire et Génique has decided to focus part of the program of the ninth annual SFTCG meeting on (1) gene therapy for the central nervous system, and (2) cell therapy and regenerative medicine.

Because several pieces of evidence have shown that chromatin instability and epigenetic changes may occur during the proliferation and differentiation of cells that have or have not been genetically modified, cell and gene therapists will also have to face important scientific questions concerning the targeting of cells, the targeting of genes of interest, the control of gene expression, and the contribution of nuclear architecture in the regulation of gene expression patterns. Thus, targeting is an increasingly important concept with the goal of precisely inserting a gene of interest at preselected sites, using homologous recombination-based strategies. Targeting and the control of genes are important priorities for the future development of cell and gene therapies. For these reasons, the SFTCG scientific board will dedicate an entire day to “targeted integration, epigenetic, and chromatin regulation.” Indeed, solving these new challenges will provide more efficient and safer tools for the development of cell and gene therapy.

As usual, the SFTCG strongly encourages the participation of young investigators by attracting them with very low registration fees and by providing prizes for the best oral and poster presentations. For the second consecutive year, the submitted abstracts will be published in Human Gene Therapy, the official journal of our society.

I hope that the SFCTG and the ninth annual meeting would respond to the expectations of our members and guests. Welcome to the 13th–15th of June, in Paris.