Engineering the next generation of cell-based therapeutics
Cell-based therapeutics are an emerging modality with the potential to treat many currently
intractable diseases through uniquely powerful modes of action. Despite notable recent …
intractable diseases through uniquely powerful modes of action. Despite notable recent …
The new frontier of genome engineering with CRISPR-Cas9
JA Doudna, E Charpentier - Science, 2014 - science.org
Background Technologies for making and manipulating DNA have enabled advances in
biology ever since the discovery of the DNA double helix. But introducing site-specific …
biology ever since the discovery of the DNA double helix. But introducing site-specific …
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …
technologies has opened up the possibility of directly targeting and modifying genomic …
Therapeutic genome editing: prospects and challenges
Recent advances in the development of genome editing technologies based on
programmable nucleases have substantially improved our ability to make precise changes …
programmable nucleases have substantially improved our ability to make precise changes …
CRISPR/Cas9 in genome editing and beyond
The Cas9 protein (CRISPR-associated protein 9), derived from type II CRISPR (clustered
regularly interspaced short palindromic repeats) bacterial immune systems, is emerging as a …
regularly interspaced short palindromic repeats) bacterial immune systems, is emerging as a …
Applications of CRISPR technologies in research and beyond
R Barrangou, JA Doudna - Nature biotechnology, 2016 - nature.com
Programmable DNA cleavage using CRISPR–Cas9 enables efficient, site-specific genome
engineering in single cells and whole organisms. In the research arena, versatile CRISPR …
engineering in single cells and whole organisms. In the research arena, versatile CRISPR …
Advances in the delivery of RNA therapeutics: from concept to clinical reality
The rapid expansion of the available genomic data continues to greatly impact biomedical
science and medicine. Fulfilling the clinical potential of genetic discoveries requires the …
science and medicine. Fulfilling the clinical potential of genetic discoveries requires the …
Diverse evolutionary roots and mechanistic variations of the CRISPR-Cas systems
BACKGROUND Prokaryotes have evolved multiple systems to combat invaders such as
viruses and plasmids. Examples of such defense systems include receptor masking …
viruses and plasmids. Examples of such defense systems include receptor masking …
[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 …
Genome-editing technologies for gene and cell therapy
ML Maeder, CA Gersbach - Molecular therapy, 2016 - cell.com
Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
However, the recent advent of genome-editing technologies has enabled a new paradigm in …