Article

Cas9 RNP Physiochemical Analysis for Enhanced CRISPR-AuNP Assembly and Function

2024-04-02

Abstract excerpt

CRISPR therapy for hematological disease has proven effective for transplant dependent beta thalassemia and sickle cell anemia, with additional disease targets in sight. The success of these therapies relies on high rates of CRISPR-induced double strand DNA breaks in hematopoietic stem and progenitor cells (HSPC). To achieve these levels, CRISPR complexes are typically delivered by electroporation ex vivo which is...

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Literature Corpus work: 6e517c74-ae0e-5b2a-b8d2-179c1d85ccf6
DOI: 10.1101/2024.04.02.586657

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