This study engineer compact photoswitchable Cas12f (psCas12f) proteins that are co-deliverable with their single guide RNA (sgRNA) in a single AAV particle, enabling precise spatiotemporal control in vivo.
Electroporation-based delivery of CRISPR/Cas systems has emerged as a powerful and versatile approach for gene editing in primary human T cells, enabling efficient, transient, and nonviral modification while minimizing genomic integration risks. This chapter focuses on the principles and practical implementation of ele...
María Ortiz-Bueno, Alejandro Millán-López, Kornel Labun et al.· Methods in molecular biology· 0 citations
Delivery remains the main obstacle to the development of in vivo genome editing therapies. CRISPR ribonucleoproteins confer high editing activity with transient exposure but lack intrinsic cell entry and targeting. Here we introduce PERCEPT, a delivery platform featuring reversible, covalent modification of CRISPR enzy...
Christopher M. Baehr, Alzbeta Ressnerova, Min Kang et al.· bioRxiv· 0 citations
Clinical applications of gene editors including CRISPR-Cas9 are limited by the cell-type specificity and tissue penetrance of current in vivo delivery options. Cell-mediated gene editor delivery could overcome these obstacles. To date, systems utilizing immortalized cell lines have demonstrated gene editor delivery in...
James Meixiong, Oleksandr Zginnyk, Lauren Chow et al.· bioRxiv· 0 citations
The rapid development of CRISPR genome editing technologies has established a transformative paradigm within biomedical research, drug discovery, and gene therapy. Despite the robust nuclease activity and programmable targeting exhibited by these systems, the clinical translation of CRISPR-mediated therapeutics remai...
Li Du, Xiao-Feng Hua, Qianquan Ma et al.· Molecular Medicine· 0 citations
Ultrasound-CRISPR technologies are elaborates potential as a powerful tool that enables targeted, and image-guided genetic interventions, capable of advancing the outcomes of gene therapy as a widespread clinical practice.
Sara Maham, B. Sridharan, J. Park et al.· Advanced Healthcare Material...· 0 citations
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