Intracellular delivery by membrane disruption: mechanisms, strategies, and concepts

MP Stewart, R Langer, KF Jensen - Chemical reviews, 2018 - ACS Publications
Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

[HTML][HTML] Mosaicism in CRISPR/Cas9-mediated genome editing

M Mehravar, A Shirazi, M Nazari, M Banan - Developmental biology, 2019 - Elsevier
The CRISPR/Cas9 system is a rapid, simple, and often extremely efficient gene editing
method. This method has been used in a variety of organisms and cell types over the past …

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

CA Vakulskas, DP Dever, GR Rettig, R Turk… - Nature medicine, 2018 - nature.com
Translation of the CRISPR–Cas9 system to human therapeutics holds high promise.
However, specificity remains a concern especially when modifying stem cell populations …

Codon-specific KRAS mutations predict survival benefit of trifluridine/tipiracil in metastatic colorectal cancer

J van de Haar, X Ma, SN Ooft, PW van der Helm… - Nature Medicine, 2023 - nature.com
Genomics has greatly improved how patients with cancer are being treated; however,
clinical-grade genomic biomarkers for chemotherapies are currently lacking. Using whole …

Easi-CRISPR: a robust method for one-step generation of mice carrying conditional and insertion alleles using long ssDNA donors and CRISPR ribonucleoproteins

RM Quadros, H Miura, DW Harms, H Akatsuka, T Sato… - Genome biology, 2017 - Springer
Background Conditional knockout mice and transgenic mice expressing recombinases,
reporters, and inducible transcriptional activators are key for many genetic studies and …

AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines

L Zhang, JA Zuris, R Viswanathan, JN Edelstein… - Nature …, 2021 - nature.com
Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits
relatively poor editing efficiency, restricting its overall utility. Here we isolate an engineered …

Easi-CRISPR for creating knock-in and conditional knockout mouse models using long ssDNA donors

H Miura, RM Quadros, CB Gurumurthy, M Ohtsuka - Nature protocols, 2018 - nature.com
CRISPR/Cas9-based genome editing can easily generate knockout mouse models by
disrupting the gene sequence, but its efficiency for creating models that require either …

Improving CRISPR–Cas specificity with chemical modifications in single-guide RNAs

DE Ryan, D Taussig, I Steinfeld… - Nucleic acids …, 2018 - academic.oup.com
CRISPR systems have emerged as transformative tools for altering genomes in living cells
with unprecedented ease, inspiring keen interest in increasing their specificity for perfectly …

The predominant PAR4 variant in individuals of African ancestry worsens murine and human stroke outcomes

F Denorme, ND Armstrong, ML Stoller… - The Journal of …, 2023 - Am Soc Clin Investig
Protease-activated receptor 4 (PAR4)(gene F2RL3) harbors a functional dimorphism,
rs773902 A/G (encoding Thr120/Ala120, respectively) and is associated with greater platelet …

[HTML][HTML] Highly efficient CRISPR-Cas9-based methods for generating deletion mutations and F0 embryos that lack gene function in zebrafish

K Hoshijima, MJ Jurynec, DK Shaw, AM Jacobi… - Developmental cell, 2019 - cell.com
Inconsistent activity limits the use of CRISPR-Cas9 in zebrafish. We show supernumerary
guanine nucleotides at the 5′ ends of single guide RNAs (sgRNAs) account for diminished …