[引用][C] Precision CRISPR for in vivo genome engineering
PC Promoter gRNA, G Edit - nature.com
Ensoma is poised to create a new therapeutic category. Leveraging first-in-class delivery
and engineering, the biotech aims to harness the power of hematopoietic stem cells (HSCs) …
and engineering, the biotech aims to harness the power of hematopoietic stem cells (HSCs) …
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 …
Transient retrovirus-based CRISPR/Cas9 all-in-one particles for efficient, targeted gene knockout
Y Knopp, FK Geis, D Heckl, S Horn, T Neumann… - … Therapy-Nucleic Acids, 2018 - cell.com
The recently discovered CRISPR/Cas9 system is widely-used in basic research and is a
useful tool for disease modeling and gene editing therapies. However, long-term expression …
useful tool for disease modeling and gene editing therapies. However, long-term expression …
Nanomedicine enables efficient CRISPR-Cas9 genome editing for disease treatment
L Ma, C Dong, M Yu, X Song, Y Yu… - Science Bulletin, 2022 - ui.adsabs.harvard.edu
In recent years, the clustered regularly interspaced short palindromic repeats-associated
protein 9 (CRISPR-Cas9) technology that won the 2020 Nobel Prize in Chemistry has …
protein 9 (CRISPR-Cas9) technology that won the 2020 Nobel Prize in Chemistry has …
Delivery of an artificial transcription regulator dCas9-VPR by extracellular vesicles for therapeutic gene activation
D Lainšček, L Kadunc, MM Keber… - ACS synthetic …, 2018 - ACS Publications
The CRISPR/Cas system has been developed as a potent tool for genome engineering and
transcription regulation. However, the efficiency of the delivery of the system into cells …
transcription regulation. However, the efficiency of the delivery of the system into cells …
Designing, packaging, and delivery of high titer CRISPR retro and lentiviruses via stereotaxic injection
CJ Fricano-Kugler, MR Williams, JR Salinaro… - JoVE (Journal of …, 2016 - jove.com
Replication defective lentiviruses or retroviruses are capable of stably integrating
transgenes into the genome of an infected host cell. This technique has been widely used to …
transgenes into the genome of an infected host cell. This technique has been widely used to …
Enhancing Safety and Efficacy of Genome Editing In Vivo with Compact Cas9 and Guide Chemical Modification
H Zhang - 2024 - repository.escholarship.umassmed …
Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins
(CRISPR/Cas) have revolutionized science and medicine. However, applying the …
(CRISPR/Cas) have revolutionized science and medicine. However, applying the …
[引用][C] 308 Ligand-free biodegradable poly (beta-amino ester) nanoparticles for targeted systemic delivery of mRNA to the lungs
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 …
Engineered virus-like particles: paving the way for effective somatic genome editing
C Zhuo, Y Tao, M Li - Signal Transduction and Targeted Therapy, 2022 - nature.com
A recent publication in Cell describes the development and application of engineered virus-
like particles (eVLPs) that efficiently package and deliver therapeutic gene-editing proteins …
like particles (eVLPs) that efficiently package and deliver therapeutic gene-editing proteins …