Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs
SA Dilliard, DJ Siegwart - Nature Reviews Materials, 2023 - nature.com
Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …
medicines that directly reprogramme the central dogma of biology to prevent and treat …
Off-target effects in CRISPR/Cas9 gene editing
C Guo, X Ma, F Gao, Y Guo - Frontiers in bioengineering and …, 2023 - frontiersin.org
Gene editing stands for the methods to precisely make changes to a specific nucleic acid
sequence. With the recent development of the clustered regularly interspaced short …
sequence. With the recent development of the clustered regularly interspaced short …
Lipid nanoparticle-mediated lymph node–targeting delivery of mRNA cancer vaccine elicits robust CD8+ T cell response
The targeted delivery of messenger RNA (mRNA) to desired organs remains a great
challenge for in vivo applications of mRNA technology. For mRNA vaccines, the targeted …
challenge for in vivo applications of mRNA technology. For mRNA vaccines, the targeted …
Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis
Safe and efficacious systemic delivery of messenger RNA (mRNA) to specific organs and
cells in vivo remains the major challenge in the development of mRNA-based therapeutics …
cells in vivo remains the major challenge in the development of mRNA-based therapeutics …
An ionizable lipid toolbox for RNA delivery
Recent years have witnessed incredible growth in RNA therapeutics, which has benefited
significantly from decades of research on lipid nanoparticles, specifically its key component …
significantly from decades of research on lipid nanoparticles, specifically its key component …
Lipids and lipid derivatives for RNA delivery
Y Zhang, C Sun, C Wang, KE Jankovic… - Chemical reviews, 2021 - ACS Publications
RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …
diseases through various mechanisms including knockdown of pathological genes …
Targeted drug delivery strategies for precision medicines
Progress in the field of precision medicine has changed the landscape of cancer therapy.
Precision medicine is propelled by technologies that enable molecular profiling, genomic …
Precision medicine is propelled by technologies that enable molecular profiling, genomic …
Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3
Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are associated with lowered
blood lipid levels, making Angptl3 an attractive therapeutic target for the treatment of human …
blood lipid levels, making Angptl3 an attractive therapeutic target for the treatment of human …
[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 …