Not exact matches
In an effort to combine the respective advantages of the AAV and CRISPR - based approaches, several groups have recently worked to enhance the
efficiency of AAV - based
gene editing via the introduction of a double strand break by using a
targeted nuclease.
Because of their unique properties such as monodispersity, large surface area and high drug loading
efficiency, silica nanoparticles (SiNPs) have been developed for a vast array of biomedical uses such as optical imaging, cancer therapy,
targeted drug delivery and controlled drug release for
genes and proteins.
We next tested the
efficiency of Cas9 / gRNAs disrupting four
genes simultaneously by co-transfecting pooled Cas9 / gRNAs
targeting MSTN, BLG, PrP and NUP
genes.
The plasmids
targeting each
gene were respectively transfected into goat fibroblasts, and their genome modification
efficiency was determined at day 3 using restriction fragment length polymorphism (RFLP) assay (see Methods).
Comparing this approach to the classic CRISPR / Cas9 system designed to cleave the DNA, the Gladstone team found that CRISPRi is actually more effective, silencing the
target gene in more than 95 percent of cells, compared with the 60 percent to 70 percent
efficiency of CRISPR / Cas9.
But CRISPR's speed,
efficiency and ability to
target multiple
genes simultaneously are making it the new standard for knockouts in mice, zebrafish and many other species.
Using ∼ 2000 Plasmodium falciparum genome sequences, we investigated the genetic diversity of eleven
gene -
targets of promising antimalarial compounds and assessed their potential
efficiency across malaria endemic regions.
Genes regulating the cerebral levels of important neurotransmitters (dopamine, serotonin, GABA, etc.) or signal transmission efficiency (neurotransmitter receptors and genes) have been targeted in association studies of major psychiatric disorders such as schizophrenia, bipolar disorders, attention deficit / hyperactivity disorder (ADHD), and autism [84], as well as of personality traits
Genes regulating the cerebral levels of important neurotransmitters (dopamine, serotonin, GABA, etc.) or signal transmission
efficiency (neurotransmitter receptors and
genes) have been targeted in association studies of major psychiatric disorders such as schizophrenia, bipolar disorders, attention deficit / hyperactivity disorder (ADHD), and autism [84], as well as of personality traits
genes) have been
targeted in association studies of major psychiatric disorders such as schizophrenia, bipolar disorders, attention deficit / hyperactivity disorder (ADHD), and autism [84], as well as of personality traits [85].