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 …
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 …
Recent progress in targeted delivery vectors based on biomimetic nanoparticles
L Chen, W Hong, W Ren, T Xu, Z Qian… - Signal transduction and …, 2021 - nature.com
Over the past decades, great interest has been given to biomimetic nanoparticles (BNPs)
since the rise of targeted drug delivery systems and biomimetic nanotechnology. Biological …
since the rise of targeted drug delivery systems and biomimetic nanotechnology. Biological …
[HTML][HTML] Engineering nanoparticle toolkits for mRNA delivery
The concept of using mRNA to produce its own medicine in situ in the body makes it an ideal
drug candidate, holding great potential to revolutionize the way we approach medicine. The …
drug candidate, holding great potential to revolutionize the way we approach medicine. The …
Engineering RNA export for measurement and manipulation of living cells
A system for programmable export of RNA molecules from living cells would enable both
non-destructive monitoring of cell dynamics and engineering of cells capable of delivering …
non-destructive monitoring of cell dynamics and engineering of cells capable of delivering …
Engineered extracellular vesicles as versatile ribonucleoprotein delivery vehicles for efficient and safe CRISPR genome editing
Transient delivery of CRISPR‐based genome editing effectors is important to reduce off‐
target effects and immune responses. Recently extracellular vesicles (EVs) have been …
target effects and immune responses. Recently extracellular vesicles (EVs) have been …
Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice
S Ling, S Yang, X Hu, D Yin, Y Dai, X Qian… - Nature biomedical …, 2021 - nature.com
Therapeutic genome editing requires effective and targeted delivery methods. The delivery
of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy …
of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy …
Engineered materials for in vivo delivery of genome-editing machinery
Genome-editing technologies, such as CRISPR–Cas9, are promising for treating otherwise
incurable genetic diseases. Great progress has been made for ex vivo genome editing; …
incurable genetic diseases. Great progress has been made for ex vivo genome editing; …
Targeted nonviral delivery of genome editors in vivo
CA Tsuchida, KM Wasko… - Proceedings of the …, 2024 - National Acad Sciences
Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that
will drive biological discovery and transform the field of cell and gene therapy. Here, we …
will drive biological discovery and transform the field of cell and gene therapy. Here, we …
[HTML][HTML] Applications and developments of gene therapy drug delivery systems for genetic diseases
X Pan, H Veroniaina, N Su, K Sha, F Jiang… - Asian journal of …, 2021 - Elsevier
Genetic diseases seriously threaten human health and have always been one of the
refractory conditions facing humanity. Currently, gene therapy drugs such as siRNA, shRNA …
refractory conditions facing humanity. Currently, gene therapy drugs such as siRNA, shRNA …