Sentences with phrase «human gene therapy treatment»
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Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the early clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof - of - concept in a second liver - mediated disease — a significant achievement in the gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»
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These human genes were also protective against alpha - synuclein - induced death, suggesting that they could be worth testing as gene therapy treatments for Parkinson's disease, Lu says.
This study represents a significant step towards the development of clinical trials in gene therapy for the curative treatment of hereditary deafness and balance loss in humans.
The SMA model pig was created using a gene therapy approach by knocking down the levels of pig SMN, followed by treatment with human SMN at early and late time points.
The research, part of a phase I clinical trial to test the safety of the treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human Gene Therapy.
The goal of the Program in Human Gene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseaGene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diTherapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseagene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseagene therapy for the treatment of genetic and acquired ditherapy for the treatment of genetic and acquired diseases.
A new animal study involving virally - delivered gene therapies has indicated that high treatment doses might not be as safe as previous human trials have suggested
«Our work in NPC1 mice may help lead to human clinical trials and eventually FDA approval for gene therapy as a treatment for NPC1 disease,» said Charles P. Venditti, M.D., Ph.D., senior investigator in the NHGRI Medical Genomics and Metabolic Genetics Branch.
Auburn University College of Veterinary Medicine, UMass Medical School to test gene therapy treatment for Tay - Sachs and Sandhoff diseases Researchers at Auburn University College of Veterinary Medicine and UMass Medical School are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.