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 …
Non-coding RNAs and potential therapeutic targeting in cancer
Recent advances have begun to clarify the physiological and pathological roles of non-
coding RNAs (ncRNAs) in various diseases, including cancer. Among these, microRNAs …
coding RNAs (ncRNAs) in various diseases, including cancer. Among these, microRNAs …
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing
Delivery technologies for the CRISPR-Cas9 (CRISPR, clustered regularly interspaced short
palindromic repeats) gene editing system often require viral vectors, which pose safety …
palindromic repeats) gene editing system often require viral vectors, which pose safety …
The great escape: how cationic polyplexes overcome the endosomal barrier
T Bus, A Traeger, US Schubert - Journal of Materials Chemistry B, 2018 - pubs.rsc.org
The targeted and efficiency-oriented delivery of (therapeutic) nucleic acids raises hope for
successful gene therapy, ie, for the local and individual treatment of acquired and inherited …
successful gene therapy, ie, for the local and individual treatment of acquired and inherited …
The delivery of therapeutic oligonucleotides
RL Juliano - Nucleic acids research, 2016 - academic.oup.com
The oligonucleotide therapeutics field has seen remarkable progress over the last few years
with the approval of the first antisense drug and with promising developments in late stage …
with the approval of the first antisense drug and with promising developments in late stage …
Direct cytosolic delivery of CRISPR/Cas9-ribonucleoprotein for efficient gene editing
Genome editing through the delivery of CRISPR/Cas9-ribonucleoprotein (Cas9-RNP)
reduces unwanted gene targeting and avoids integrational mutagenesis that can occur …
reduces unwanted gene targeting and avoids integrational mutagenesis that can occur …
Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities
L Li, S Hu, X Chen - Biomaterials, 2018 - Elsevier
In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas
(CRISPR-associated) genome editing systems have become one of the most robust …
(CRISPR-associated) genome editing systems have become one of the most robust …
Application of CRISPR/Cas9-based gene editing in HIV-1/AIDS therapy
Q Xiao, D Guo, S Chen - Frontiers in cellular and infection …, 2019 - frontiersin.org
Despite the fact that great efforts have been made in the prevention and therapy of HIV-1
infection, HIV-1/AIDS remains a major threat to global human health. Highly active …
infection, HIV-1/AIDS remains a major threat to global human health. Highly active …
Strategies for nonviral nanoparticle‐based delivery of CRISPR/Cas9 therapeutics
CRISPR‐based genome editing technology has become an important potential therapeutic
tool for various diseases. A vital challenge is to reach a safe, efficient, and clinically suitable …
tool for various diseases. A vital challenge is to reach a safe, efficient, and clinically suitable …
Macrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles
The CRISPR/Cas9 gene editing technology holds promise for the treatment of multiple
diseases. However, the inability to perform specific gene editing in targeted tissues and …
diseases. However, the inability to perform specific gene editing in targeted tissues and …