Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
Lipid nanoparticle (LNP) enables mRNA delivery for cancer therapy
Messenger RNA (mRNA) has received great attention in the prevention and treatment of
various diseases due to the success of coronavirus disease 2019 (COVID‐19) mRNA …
various diseases due to the success of coronavirus disease 2019 (COVID‐19) mRNA …
On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles
SA Dilliard, Q Cheng… - Proceedings of the …, 2021 - National Acad Sciences
Lipid nanoparticles (LNPs) are a clinically mature technology for the delivery of genetic
medicines but have limited therapeutic applications due to liver accumulation. Recently, our …
medicines but have limited therapeutic applications due to liver accumulation. Recently, our …
Enhancing CRISPR/Cas gene editing through modulating cellular mechanical properties for cancer therapy
Genome editing holds great potential for cancer treatment due to the ability to precisely
inactivate or repair cancer-related genes. However, delivery of CRISPR/Cas to solid tumours …
inactivate or repair cancer-related genes. However, delivery of CRISPR/Cas to solid tumours …
Application of the CRISPR/Cas9-based gene editing technique in basic research, diagnosis, and therapy of cancer
H Zhang, C Qin, C An, X Zheng, S Wen, W Chen, X Liu… - Molecular cancer, 2021 - Springer
Abstract The 2020 Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and
Jennifer Doudna for the development of the Clustered regularly interspaced short …
Jennifer Doudna for the development of the Clustered regularly interspaced short …
[HTML][HTML] Advances in CRISPR therapeutics
The clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …
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 …
Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery
A new methodology termed selective organ targeting (SORT) was recently developed that
enables controllable delivery of nucleic acids to target tissues. SORT lipid nanoparticles …
enables controllable delivery of nucleic acids to target tissues. SORT lipid nanoparticles …
Zwitterionic phospholipidation of cationic polymers facilitates systemic mRNA delivery to spleen and lymph nodes
Polymers represent a promising therapeutic platform for extrahepatic messenger RNA
(mRNA) delivery but are hampered by low in vivo efficacy due to polyplex serum instability …
(mRNA) delivery but are hampered by low in vivo efficacy due to polyplex serum instability …
[HTML][HTML] In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
Within less than a decade since its inception, CRISPR-Cas9-based genome editing has
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …