Sentences with phrase «gene therapy approaches studied»

Gene Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decade.

Not exact matches

Indeed, exposure of the protein produced by the nanoparticle - based gene therapy to the gut mucosa prevents inhibitor development and restores clotting - factor activity in mouse models of both haemophilia A and B. «This approach really could hold big benefit for patients,» says Jörg Schüttrumpf, a transfusion - medicine specialist who led one of the studies performed at the German Red Cross Blood Donor Service in Frankfurt.

A study published January 4th in Cell Stem Cell demonstrates that a gene therapy approach can lead to the long - term survival of functional beta cells as well as normal blood glucose levels for an extended period of time in mice with diabetes.

Field reports suggest that not all K13 mutations are capable of causing resistance, and the genetic system developed by Dr. Fidock to study K13, based on DNA repair approaches that are being used in human gene therapy studies, will be critical in identifying real hot spots of resistance.

«We recognize that there are many other approaches [to pay for gene therapy] that thoughtful study might uncover,» the authors conclude, «but we need to begin to ensure that economic challenges are given the attention they deserve.»

Although the study's approach wasn't a sure bet, the researchers hoped to go further than any other gene therapy trial yet for this relatively common inherited disease that fills people's lungs with sticky mucus that promotes deadly infections.

«This study suggests that targeting p27, GATA3 and POU4F3 may enhance the outcome of gene therapy and other approaches that aim to restart ATOH1 expression,» Zuo said.

«Efficacy of new gene therapy approach for toxin exposures shown in mouse study.»

PNP Therapeutics, Inc. — a company based on a gene - therapy technology developed by scientists at Southern Research Institute and the University of Alabama at Birmingham (UAB)-- has found an approach that has abolished otherwise unmanageable human cancers in preclinical rodent studies.

«In our study, we used a gene therapeutic approach, but you could purify the modified protein and inject it intravenously just like it is already done for enzyme replacement therapy,» explains Spencer.

And he should know - Wilson has been studying CF for over 20 years, as well as many other rare diseases and gene - therapy approaches to treat them.

This approach may also be applicable to a number of rare genetic diseases caused by splicing defects, including ataxia telangiectasia, congenital disorder of glycosylation, and Niemann - Pick disease type C. Although the three DMD studies referenced here represent a great step forward for CRISPR gene therapy, it's important to realize that DMD is a simpler case than other genetic diseases we'd like to treat with CRISPR.

Lysogene collaborates with University of Massachusetts Medical School (UMMS) for pre-clinical efficacy studies of innovative AAV - based gene therapy approaches in the GM1 gangliosidosis mouse model.

Williams and his colleagues are studying a different approach to sickle cell gene therapy.