Genome-editing technologies: principles and applications

T Gaj, SJ Sirk, S Shui, J Liu - Cold Spring Harbor …, 2016 - cshperspectives.cshlp.org
Targeted nucleases have provided researchers with the ability to manipulate virtually any
genomic sequence, enabling the facile creation of isogenic cell lines and animal models for …

Rational design of nanocarriers for intracellular protein delivery

X Qin, C Yu, J Wei, L Li, C Zhang, Q Wu… - Advanced …, 2019 - Wiley Online Library
Protein/antibody therapeutics have exhibited the advantages of high specificity and activity
even at an extremely low concentration compared to small molecule drugs. However, they …

Nanotechnology for protein delivery: Overview and perspectives

M Yu, J Wu, J Shi, OC Farokhzad - Journal of controlled release, 2016 - Elsevier
Protein-based therapeutics have made a significant impact in the treatment of a variety of
important human diseases. However, given their intrinsically vulnerable structure and …

Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells

J Liu, T Gaj, Y Yang, N Wang, S Shui, S Kim… - Nature protocols, 2015 - nature.com
Targeted nucleases, including zinc-finger nucleases (ZFNs), transcription activator-like
(TAL) effector nucleases (TALENs) and clustered regularly interspaced short palindromic …

Trapped! A critical evaluation of methods for measuring total cellular uptake versus cytosolic localization

K Deprey, L Becker, J Kritzer… - Bioconjugate …, 2019 - ACS Publications
Biomolecules have many properties that make them promising for intracellular therapeutic
applications, but delivery remains a key challenge because large biomolecules cannot …

Synthetic Peptides: Promising Modalities for the Targeting of Disease‐Related Nucleic Acids

BD Ellenbroek, JP Kahler, SR Evers… - Angewandte …, 2024 - Wiley Online Library
DNA and RNA play pivotal roles in life processes by storing and transferring genetic
information, modulating gene expression, and contributing to essential cellular machinery …

Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery

T Gaj, BT Staahl, GMC Rodrigues… - Nucleic acids …, 2017 - academic.oup.com
Realizing the full potential of genome editing requires the development of efficient and
broadly applicable methods for delivering programmable nucleases and donor templates for …

Acid‐Labile Acyclic Cucurbit[n]uril Molecular Containers for Controlled Release

D Mao, Y Liang, Y Liu, X Zhou, J Ma… - Angewandte Chemie …, 2017 - Wiley Online Library
Stimuli‐responsive molecular containers are of great importance for controlled drug delivery
and other biomedical applications. A new type of acid labile acyclic cucurbit [n] uril (CB [n]) …

Boronic Acid-Linked Cell-Penetrating Peptide for Protein Delivery

P Ghosh - ACS omega, 2024 - ACS Publications
Studying functional protein delivery into live cells is important, ranging from fundamental
research to therapeutics. Cell-penetrating peptides (CPPs) are known to deliver proteins …

Potent and selective inhibition of pathogenic viruses by engineered ubiquitin variants

W Zhang, BA Bailey-Elkin, RCM Knaap, B Khare… - PLoS …, 2017 - journals.plos.org
The recent Middle East respiratory syndrome coronavirus (MERS-CoV), Ebola and Zika
virus outbreaks exemplify the continued threat of (re-) emerging viruses to human health …