Sentences with phrase «gene correction»
«This embryo gene correction method — if proven safe — can potentially be used to prevent transmission of genetic disease to future generations,» says study co-author Paula Amato.
discovermagazine.com
«This embryo gene correction method, if proven safe, can potentially be used to prevent transmission of genetic disease to future generations,» Amato said.
Not only did a high percentage of embryonic cells get repaired, but also gene correction didn't induce any detectable off - target mutations and genome instability — major concerns for gene editing.
Researchers describe how a new «universal» strategy for CRISPR - Cas9 - mediated gene correction in patient derived stem cells may represent a promising therapeutic option
Seamless gene correction of β - thalassemia mutations in patient - specific iPSCs using CRISPR / Cas9 and piggyBac.
Using gene correction, we have shown that one of these mutations — LRRK2 G2019S — causes PD through a novel mechanism.
A significant focus of the Structural Biology Group is the exploitation of structural information to engineer macromolecules for therapeutic applications, including gene correction, vaccine development, and drug discovery.
«If you have a gene correction strategy,» he says, «you'd better have these primitive epidermal stem cells in mind.»
«Even though the success rate in patient cells cultured in a dish was low, we saw that the gene correction seems to be very robust in embryos of which one copy of the MYBPC3 gene is mutated,» says Jun Wu, a Salk staff scientist and one of the paper's first authors.
To successfully treat genetic blood diseases like SCID, however, researchers first have to show that the scissor method works on the blood cell - producing hematopoietic stem cells — required for a lasting cure — which Kohn says «may be less susceptible to gene correction, and may be more fragile to treat.»
Adds Thomson: «In this new approach, Dr. Howden succeeded in combining the reprogramming and the gene correction steps together using the new Cas9 / CRISPR technology, greatly reducing the time required.»