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Papers on “CRISPR gene editing therapeutic applications”

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1. Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

   Ophir Shalem, Neville E. Sanjana, Ella Hartenian, et al. · 2013 · Science · 5,608 cites

   The simplicity of programming the CRISPR (clustered regularly interspaced short palindromic repeats)-associated nuclease Cas9 to modify specific genomic loci suggests a new way to interrogate gene function on a genome-wide scale. We show that lentiviral delivery of a genome-scale CRISPR-Cas9 knockout (GeCKO) library targeting 18,080 genes with 64,751 unique guide sequences enables both negative and positive selection screening in human cells. First, we used the GeCKO library to identify genes essential for cell viability in cancer and pluripotent stem cells. Next, in a melanoma model, we screened for genes whose loss is involved in resistance to vemurafenib, a therapeutic RAF inhibitor. Our

2. In vivo genome editing using Staphylococcus aureus Cas9

   F. Ann Ran, Le Cong, Winston X. Yan, et al. · 2015 · Nature · 2,763 cites

3. High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

   Benjamin P. Kleinstiver, Vikram Pattanayak, Michelle S. Prew, et al. · 2016 · Nature · 2,748 cites

4. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

   Julian D. Gillmore, Ed Gane, Jörg Täubel, et al. · 2021 · New England Journal of Medicine · 1,721 cites

   BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of the two initial dose groups (0.1 mg per kilogram and 0.3 mg per kilogram), within an ongoing phase 1 clinical study. RESULTS: after a single dose. Serial assessments of safety during the first 28 days after infusion in patients revealed few adverse events, and those that did occur were mild in grade. Dose-dependent pharmacodynamic effects were observed. At day 28, the mean reduction from baseline in serum TTR protein concentration was

5. The CRISPR tool kit for genome editing and beyond

   Mazhar Adli · 2018 · Nature Communications · 1,684 cites

   CRISPR is becoming an indispensable tool in biological research. Once known as the bacterial immune system against invading viruses, the programmable capacity of the Cas9 enzyme is now revolutionizing diverse fields of medical research, biotechnology, and agriculture. CRISPR-Cas9 is no longer just a gene-editing tool; the application areas of catalytically impaired inactive Cas9, including gene regulation, epigenetic editing, chromatin engineering, and imaging, now exceed the gene-editing functionality of WT Cas9. Here, we will present a brief history of gene-editing tools and describe the wide range of CRISPR-based genome-targeting tools. We will conclude with future directions and the broa

6. CRISPR/Cas9 in Genome Editing and Beyond

   Haifeng Wang, Marie La Russa, Lei S. Qi · 2016 · Annual Review of Biochemistry · 1,213 cites

   The Cas9 protein (CRISPR-associated protein 9), derived from type II CRISPR (clustered regularly interspaced short palindromic repeats) bacterial immune systems, is emerging as a powerful tool for engineering the genome in diverse organisms. As an RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence, and its development as a tool has made sequence-specific gene editing several magnitudes easier. The nuclease-deactivated form of Cas9 further provides a versatile RNA-guided DNA-targeting platform for regulating and imaging the genome, as well as for rewriting the epigenetic status, all in a sequence-specific manner. With all of these

7. In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

   Keiichiro Suzuki, Yuji Tsunekawa, Reyna Hernández‐Benítez, et al. · 2016 · Nature · 1,201 cites

8. A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

   Jonathan D. Finn, Amy Smith, Mihir Patel, et al. · 2018 · Cell Reports · 875 cites

   The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The