Sentences with phrase «clinical gene therapy applications»

Not exact matches

«While much work remains to be done to develop this research into clinical applications, our findings could have a promising translational impact, both for protein replacement and gene therapy,» said Arruda.

There already has been great effort to elucidate the mechanisms of gene expression regulation, but it will be important to continue research to fully realize the potential of these epigenetic therapies and their applications in clinical settings.

It could also be relevant to clinical applications — it treats sequence uniqueness as a high priority and thus minimises the risk of potentially unwanted gene modifications, which must be avoided at all costs in gene therapy,» says Graf.

«Having a very efficient and practical way of generating patient - specific stem cells, which unlike human embryonic stem cells, wouldn't be rejected by the patient's immune system after transplantation brings us a step closer to the clinical application of stem cell therapy,» says Belmonte, PhD., a professor in the Gene Expression Laboratory and director of the Center of Regenerative Medicine in Barcelona, Spain.

Clinical Applications and Bioprocessing Strategies Fundamentally, gene therapy should exceed the efficacy of existing therapeutic approaches and provide therapeutic strategies for unmet medical needs.

For many years, gene therapy had shown great promise, but clinical applications and their impact upon patient lives always appeared out of reach.

Among other accomplishments, this milestone - based program made possible the development and clinical testing of antisense therapy against a common gene mutation in ALS, the first application of antisense therapy to any neurologic disease.