Sentences with phrase «gene replacement therapy»

Indeed, Leber Congenital Amaurosis (LCA) was one of the first recessive disorders to undergo clinical trials for gene replacement therapy.

Gene therapy is a promising approach for treating a large spectrum of human diseases and particularly monogenic diseases that can be corrected through gene replacement therapy based on introducing a functional copy of the defective gene.

Study using disabled form of HIV in gene therapy wins highest award from Clinical Research Forum; others cited for advances revealing damage caused by long - term cortisone shots to treat arthritic knees; and gene replacement therapy for infants with deadly neuromuscular disease

He tested, for the first time in history, intravenous gene replacement therapy for a fatal disease in infants, spinal muscular atrophy.

The SMA gene replacement therapy developed by the Mendell and Kaspar teams at Nationwide Children's Hospital and collaborators at Ohio State University has the realistic potential to cure a devastating neurological disorder of newborn infants.

«Encouraging data for gene replacement therapy for SMA type I, phase 1 study shows.»

Not exact matches

The finding offers potential for developing both gene therapy and more effective protein replacement treatments for hemophilia A, the most common form of hemophilia.

«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's no way to cram the dystrophin gene into a virus to do traditional replacement gene therapy, but researchers have found that turning on other genes can compensate and bulk up muscles.

2009: A resounding success brings gene therapy back into the limelight: Jean Bennett and Albert Maguire announce that 12 patients with a degenerative disorder leading to total blindness were treated with viruses bearing a replacement gene, and all regain some vision.

One potential treatment for CF is gene therapy, and a major challenge in gene therapy is packaging replacement genes so they can be delivered to the target cells.

Tina Hesman Saey covers the ethics of gene editing, and Aimee Cunningham revisits hormone replacement therapy.

Hormone ‐ replacement therapy influences gene expression profiles and is associated with breast ‐ cancer prognosis: a cohort study

Basically what was happening when you use a hematopoietic stem cell to correct an inherited metabolic disease is that through engraftment of that cell you are allowing that cell to become the replacement source for the missing enzyme or other factor - almost like a cellular form of gene therapy or, as I call it, «poor man's gene therapy».

«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.

The title of the paper is «The Ideal Gene Delivery Vector: Chromallocytes, Cell Repair Nanorobots for Chromosome Replacement Therapy» and it is currently in press at the peer - reviewed Journal of Evolution and Technology (and is soon to be available online).

Tags: bone marrow transplantation, Clinical Research, D. Gary Gilliland, Effie Petersdorf, Garnet Anderson, gregory fund, Harlan Robins, hla gene, hormone replacement therapy, Human Immunogenetics, jamie moyer, Jason Bielas, Kiem Lab, Moyer Foundation, Public Health Sciences, Women's Health Initiative Clinical Coordinating Center

December 6, 2016 — A targeted gene - replacement technique partially restored vision in blind rats and holds promise as a potential tool for gene therapy.

Hereditary diseases like SMA maybe a prime therapeutic indications for gene therapy, where gene therapy will provide a functional replacement for a dysfunctional gene.

«We are really excited about this work because, short of correcting a faulty gene, protein - replacement therapy using mRNA is one of the most promising techniques we have at our disposal,» says senior author Inder Verma, professor of genetics and holder of Salk's Irwin and Joan Jacobs Chair in Exemplary Life Science.

Moreover, while still uncommonly used in the clinic, researchers have successfully developed protein replacement therapy and gene therapy for a number of coagulopathies.

Experimentally, gene therapy, bone marrow transplants and enzyme replacement seem to have helped some.