Promising results with new gene therapy approach for treating inherited neurodegenerative diseases : eurekalert

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Promising results with new gene therapy approach for treating inherited neurodegenerative diseases

Promising results with new gene therapy approach for treating inherited neurodegenerative diseases

New Rochelle, NY, July 28, 2016-- A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold.The implications of these promising results for developing similar gene therapies for use in hu-mans and for targeting additional brain disorders are discussed in two articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.The articles are part of a special issue on CNS disorders and are available free for download on the Human Gene Therapy website until August 28, 2016.


besides news-medical

Study verifies human gene therapy in MPS I animal model

Study verifies human gene therapy in MPS I animal model

Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I.It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells.MPS I eventually leads to the abnormal accumulation of sugar debris and cell death.


moreover from biopharmadive

Phase 1 trial reveals high efficacy for hemophilia A gene therapy

Phase 1 trial reveals high efficacy for hemophilia A gene therapy

Dive Brief:The hemophilia A community received good news on Wednesday when BioMarin announced the results of a phase 1 trial, which showed that its gene therapy may have cured six of the nine participants of their hemophilia A symptoms by increasing plasma factor VIII (FVIII) levels to the normal level of 50%.Patients with severe symptoms register FVIII levels under 1%.Hemophilia A patients suffer from deficiency of the FVIII clotting protein, which leads to increased bleeding periods that can have a destructive impact on joints and diminish quality of life, according to the National Hemophilia Foundation.