Alessia Cavazza, PhD, of Great Ormond Street Institute of Child Health (ICH) at University College London, discusses her research related to the potential of emerging gene-editing technologies, particularly CRISPR, to treat primary immunodeficiency. Her work shows that CRISPR may lead to comparable or even better results than lentiviral therapy. Dr Cavazza notes that they anticipate beginning phase 1/2 clinical trials within 3 to 5 years.
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Reference: O'Hanlon Cohrt K. CRISPR-Based Correction of Rare Immune Disorder, Wiskott-Aldrich Syndrome – Interview with Alessia Cavazza. Published September 14, 2020. Accessed February 9, 2021. https://crisprmedicinenews.com/news/crispr-based-correction-of-rare-immune-disorder-wiskott-aldrich-syndrome-interview-with-alessia-c/