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 …
The promise and challenge of therapeutic genome editing
JA Doudna - Nature, 2020 - nature.com
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …
cell fates and organism traits, has the potential to both improve our understanding of human …
[HTML][HTML] Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
Replacing or editing disease-causing mutations holds great promise for treating many
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …
Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing
CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold
tremendous potential to effectively treat disease-causing mutations with diverse cellular …
tremendous potential to effectively treat disease-causing mutations with diverse cellular …
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 …
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 …
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 …
The next generation of CRISPR–Cas technologies and applications
A Pickar-Oliver, CA Gersbach - Nature reviews Molecular cell biology, 2019 - nature.com
The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …
RNA drugs and RNA targets for small molecules: principles, progress, and challenges
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …
Adeno-associated virus vector as a platform for gene therapy delivery
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …
treatment of a variety of human diseases. Recent advances in developing clinically …