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BG147 promises to transform ex vivo and in vivo gene therapies by transiently inhibiting intrinsic immune barriers mediated by IFITM3 to enhance a wide range of protocols.
A clinical-compatible platform enabling large-scale manufacturing while preserving HSPC viability, stemness, and multilineage functionality is established and a robust and adaptable roadmap for future HDR-based gene editing platforms is offered.
A comprehensive review of delivery modalities of CRISPR systems solely in vivo that underpin their therapeutic translation and outlines the remaining barriers to durable, tissue-selective, and broadly deployable CRISPR therapeutics is provided.
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