Sentences with phrase «based gene delivery»

We have established a safe and efficient method of inducing transient (removable) pluripotency based upon state - of - the - art transposon - based gene delivery vectors.

The variety of new tools available for genetic manipulation now include lentiviral - based gene delivery, and gene editing using CRISPR / Cas9, zinc finger nucleases (ZFNs) or transcription activator - like effector nucleases (TALENs).

Not exact matches

Now, to enable widespread gene delivery throughout the central and peripheral nervous systems, Caltech researchers have developed two new variants of a vector based on an adeno - associated virus (AAV): one that can efficiently ferry genetic cargo past the blood - brain barrier; and another that is efficiently picked up by peripheral neurons residing outside the brain and spinal cord, such as those that sense pain and regulate heart rate, respiration, and digestion.

The advent of molecular cloning, DNA sequencing and the many tools of molecular genetics and cell biology has given us sufficient knowledge of the basis for disease and the genes to target, but what has limited the application of gene therapy has been efficient gene delivery systems.

To address the editing challenges, we have developed a system that is based on the delivery of recombinant Cas9 protein complexed with an sgRNA targeting the gene of interest (Cas9 / sgRNA ribonucleoproteins [RNPs]-RRB- via cell - derived nanovesicles, called gesicles.

[6] Bioplex technology: Novel synthetic gene delivery system based on peptides anchored to nucleic acids.

These features enable gesicles to knock out genes with high efficiency and in a broader range of cell types than plasmid - based delivery methods.

We have demonstrated that our liposomes can mediate in vivo target mRNA delivery as well as gene disruption using an entirely RNA - based approach.

Given the tailored inhibition of selected genes and the added precision brought by targeted delivery systems, RNAi - based therapies are thought to carry lower risk of failure than traditional approaches as the biological effects are more predictable.

Researchers describe how a bacterial infection - based protein delivery strategy can mediate effective and safe gene editing in human pluripotent stem cells.

The first step in both direct and cell - based gene therapy is packaging a therapeutic transgene into a delivery vehicle (e.g., a viral vector), followed by expansion of its host cell lines to produce high - enough vector concentrations.