CRISPR technology: A decade of genome editing is only the beginning
JY Wang, JA Doudna - Science, 2023 - science.org
The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome
editing, coupled with advances in computing and imaging capabilities, has initiated a new …
editing, coupled with advances in computing and imaging capabilities, has initiated a new …
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 …
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …
advantages over nucleic acid delivery approaches. We report the development and …
Exosome-mediated delivery of Cas9 ribonucleoprotein complexes for tissue-specific gene therapy of liver diseases
CRISPR-Cas9 gene editing has emerged as a powerful therapeutic technology, but the lack
of safe and efficient in vivo delivery systems, especially for tissue-specific vectors, limits its …
of safe and efficient in vivo delivery systems, especially for tissue-specific vectors, limits its …
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading
J Rädler, D Gupta, A Zickler, SEL Andaloussi - Molecular Therapy, 2023 - cell.com
Extracellular vesicles (EVs) are gaining increasing attention for diagnostic and therapeutic
applications in various diseases. These natural nanoparticles benefit from favorable safety …
applications in various diseases. These natural nanoparticles benefit from favorable safety …
Drug delivery systems for CRISPR-based genome editors
V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice
E Kenjo, H Hozumi, Y Makita, KA Iwabuchi… - Nature …, 2021 - nature.com
Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …
[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 …
Engineered extracellular vesicles for cancer therapy
X Zhang, H Zhang, J Gu, J Zhang, H Shi… - Advanced …, 2021 - Wiley Online Library
Extracellular vesicles (EVs) have emerged as a novel cell‐free strategy for the treatment of
many diseases including cancer. As a result of their natural properties to mediate cell‐to‐cell …
many diseases including cancer. As a result of their natural properties to mediate cell‐to‐cell …
[HTML][HTML] Chondrocyte-specific genomic editing enabled by hybrid exosomes for osteoarthritis treatment
Y Liang, X Xu, L Xu, Z Iqbal, K Ouyang, H Zhang… - Theranostics, 2022 - ncbi.nlm.nih.gov
Rationale: A cell-specific delivery vehicle is required to achieve gene editing of the disease-
associated cells, so the hereditable genome editing reactions are confined within these cells …
associated cells, so the hereditable genome editing reactions are confined within these cells …