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 …
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 …
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
We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling,
noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe …
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
CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …
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 …
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 …
tumors that is related to their particular anatomical and pathophysiological characteristics …
[HTML][HTML] Nanotechnology-based delivery of CRISPR/Cas9 for cancer treatment
Abstract CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats-
associated protein 9) is a potent technology for gene-editing. Owing to its high specificity …
associated protein 9) is a potent technology for gene-editing. Owing to its high specificity …
Recent advancements in liposome technology
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 …
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 …
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
Abstract Approximately 10% of Cystic Fibrosis (CF) patients, particularly those with CF
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …