New gene therapy corrects a form of inherited macular degeneration in canine model

(University of Pennsylvania) Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable.
Source: EurekAlert! - Biology - Category: Biology Source Type: news

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Use of Precision-Cut Lung Slices as an Ex Vivo Tool for Evaluating Viruses and Viral Vectors for Gene and Oncolytic Therapy. Mol Ther Methods Clin Dev. 2018 Sep 21;10:245-256 Authors: Rosales Gerpe MC, van Vloten JP, Santry LA, de Jong J, Mould RC, Pelin A, Bell JC, Bridle BW, Wootton SK Abstract Organotypic slice cultures recapitulate many features of an intact organ, including cellular architecture, microenvironment, and polarity, making them an ideal tool for the ex vivo study of viruses and viral vectors. Here, we describe a procedure for generating precision-cut ovine and murine tissue ...
Source: Clinical Lung Cancer - Category: Cancer & Oncology Authors: Tags: Mol Ther Methods Clin Dev Source Type: research
Conditions:   Relapsed Neuroblastoma;   Refractory Neuroblastoma Interventions:   Genetic: C7R-GD2.CART cells;   Drug: Cyclophosphamide;   Drug: Fludarabine Sponsors:   Baylor College of Medicine;   Texas Children's Hospital;   Center for Cell and Gene Therapy, Baylor College of Medicine Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
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Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Medscape Today News Source Type: news
Will Woleben, six, from McKinney, Texas, was diagnosed with Leigh syndrome in April 2014. Now his parents are working to raise $300,000 to enroll him in a gene therapy trial.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
THURSDAY, Aug. 16, 2018 -- A U.S. National Institutes of Health oversight panel will no longer review all applications for gene therapy experiments, according to a perspective piece published online Aug. 15 in the New England Journal of...
Source: Drugs.com - Pharma News - Category: Pharmaceuticals Source Type: news
The agency proposes ceding its scrutiny of these studies to the FDA.
Source: The Scientist - Category: Science Tags: News & Opinion Source Type: news
Conditions:   Alzheimer Disease;   Early Onset Alzheimer Disease Intervention:   Biological: AAVrh.10hPOE2 vector Sponsor:   Weill Medical College of Cornell University Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Here are some of the latest health and medical news developments, compiled by the editors of HealthDay: NIH Panel Will No Longer Review Gene Therapy Experiments A U.S. National Institutes of Health oversight panel will no longer review all...
Source: Drugs.com - Daily MedNews - Category: General Medicine Source Type: news
No abstract available
Source: Neurology Today - Category: Neurology Tags: Features Source Type: research
by Miguel A. Mu ñoz-Lorente, Paula Martínez, Águeda Tejera, Kurt Whittemore, Ana Carolina Moisés-Silva, Fàtima Bosch, Maria A. Blasco Short and dysfunctional telomeres are sufficient to induce a persistent DNA damage response at chromosome ends, which leads to the induction of senescence and/or apoptosis and to various age-related conditions, including a group of diseases known as “telomere syndromes”, which are provoked by extremely short telomeres owing to germline mutations in telomere genes. This opens the possibility of using telomerase activation as a potential therapeu...
Source: PLoS Genetics - Category: Genetics & Stem Cells Authors: Source Type: research
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