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Alternatives to CRISPR-Cas9 address limitations like delivery challenges and off-target effects. Emerging nucleases offer improved specificity, smaller sizes, and therapeutic applications.
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Incorporating novel nucleases into CRISPR therapies offers significant IP advantages, transforming therapeutic development. Unlike traditional licensing agreements, novel nucleases simplify financial terms, reducing negotiation complexities and encouraging innovation
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Explore the intricacies of Chemistry, Manufacturing, and Controls (CMC) requirements and Critical Quality Attributes (CQA), for CRISPR-based therapeutic development including how they differ from traditional drug and therapy development.
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Chemical modifications on CRISPR gRNAs are essential for enhancing stability, reducing immune responses, and improving editing efficiency. Learn how these modifications act as protective armor, preventing degradation by exonucleases and stabilizing gRNAs.
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A crucial consideration for researchers is off-target editing — unintended DNA modifications at sites other than the intended target. Explore the complexities and strategies to mitigate these effects.
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