Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

[HTML][HTML] Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

S Zhang, J Shen, D Li, Y Cheng - Theranostics, 2021 - ncbi.nlm.nih.gov
CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy

Y Zou, X Sun, Q Yang, M Zheng, O Shimoni… - Science …, 2022 - science.org
We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling,
noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe …

[HTML][HTML] Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

T Wei, Q Cheng, YL Min, EN Olson… - Nature communications, 2020 - nature.com
CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …

Polymeric delivery of therapeutic nucleic acids

R Kumar, CF Santa Chalarca, MR Bockman… - Chemical …, 2021 - ACS Publications
The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …

Exploiting the dynamics of the EPR effect and strategies to improve the therapeutic effects of nanomedicines by using EPR effect enhancers

J Fang, W Islam, H Maeda - Advanced drug delivery reviews, 2020 - Elsevier
The enhanced permeability and retention (EPR) effect is a unique phenomenon of solid
tumors that is related to their particular anatomical and pathophysiological characteristics …

[HTML][HTML] Nanotechnology-based delivery of CRISPR/Cas9 for cancer treatment

X Xu, C Liu, Y Wang, O Koivisto, J Zhou, Y Shu… - Advanced drug delivery …, 2021 - Elsevier
Abstract CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats-
associated protein 9) is a potent technology for gene-editing. Owing to its high specificity …

Recent advancements in liposome technology

N Filipczak, J Pan, SSK Yalamarty… - Advanced drug delivery …, 2020 - Elsevier
The liposomes have continued to be well-recognized as an important nano-sized drug
delivery system with attractive properties, such a characteristic bilayer structure assembling …

[HTML][HTML] A comprehensive review on novel liposomal methodologies, commercial formulations, clinical trials and patents

VVSNL Andra, SVN Pammi, LVKP Bhatraju… - Bionanoscience, 2022 - Springer
Liposomes are well-recognized and essential nano-sized drug delivery systems. Liposomes
are phospholipid vesicles comprised of cell membrane components and have been …

[HTML][HTML] Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models

T Wei, Y Sun, Q Cheng, S Chatterjee, Z Traylor… - Nature …, 2023 - nature.com
Abstract Approximately 10% of Cystic Fibrosis (CF) patients, particularly those with CF
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …