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Sickle Cell Therapy

All articles tagged with #sickle cell therapy

science-and-technology1 year ago

"Unlocking the Potential: Jennifer Doudna's Insights on Gene Editing"

Nobel laureate Jennifer Doudna, a pioneer of CRISPR-Cas9 gene editing, emphasizes the need for more efficient and affordable delivery methods for CRISPR-based therapies, following the recent FDA approval of a sickle-cell therapy. The current process, involving bone marrow extraction and genetic engineering, is physically challenging and costly, limiting its widespread use. Doudna advocates for in vivo genome editing to address this bottleneck, highlighting ongoing efforts to develop nanoparticle delivery systems. She remains confident that solving the delivery challenge will unlock the immense potential of gene editing to alleviate human suffering.