(CercleFinance.com) – Cellectisa today announced the publication of an article in Molecular Therapy – Methods & Clinical Development, demonstrating the effectiveness of its TALEN® genome editing platform in editing the mutated PIK3CD gene in APDS1 T cells.
APDS1, or ‘activated phosphoinositide 3-kinase δ syndrome’ is a rare but devastating disease caused by mutations in the PIK3CD gene that result in combined immunodeficiency.
Until now, there are no optimal or long-term therapeutic solutions for patients with APDS1 and new alternative treatments are highly desired, emphasizes the laboratory.
The published study evaluates the therapeutic strategy to correct the dominant mutation of the PIK3CD gene associated with APDS1 by gene editing.
‘The results show an efficient insertion of the gene into the T cells of APDS1 patients, a normalization of PI3K signaling and a restoration of the cytotoxic functions of the T cells,’ says the laboratory.
“This study paves the way for the future development of a ‘bona fide’ gene surgery candidate to potentially cure APDS1,” said Julien Valton, Ph.D., vice president of gene therapy at Cellectis.
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