One Step Closer To Gene Therapy For Cystic Fibrosis

4 November 1996

Targeted Genetics of Seattle, USA, has announced the results of two Phase I trials of AAV-CFTR, a gene therapy for cystic fibrosis, and concludes that the approach is safe, and can achieve efficient gene transfer and long-term gene persistence.

TG's approach makes use of a viral vector, adeno-associated virus, which has never been associated with human disease. The AAV-CFTR product comprises genetically-modified AAV which is non-replicating and carries the gene for cystic fibrosis transmembrane receptor, a protein which is dysfunctional in CF patients.

Persistent Gene Expression The company reports that expression times for the CFTR protein were as long as 70 days in one patient, significantly higher than has been achieved with other vectors. In addition, and in contrast to studies using adenoviral vectors (Marketletters passim), the AAV-CFTR gene product has not been associated with any inflammatory effects or other side effects, even after repeat delivery, according to Richard Daifuku, vice president of clinical affairs at TG.

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