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FDA Panel Considers Gene Therapy for Hereditary Eye Disease

Title: FDA Panel Considers Gene Therapy for Hereditary Eye DiseaseCategory: Health NewsCreated: 10/10/2017 12:00:00 AMLast Editorial Review: 10/11/2017 12:00:00 AM
Source: MedicineNet Eyesight General - Category: Opthalmology Source Type: news

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Contributors : Juan M Povedano ; Paula Martinez ; Gonzalo Gomez-Lopez ; Maria A BlascoSeries Type : Expression profiling by arrayOrganism : Mus musculusIn our previous study, mice with pulmonary fibrosis induced by a bleomycin insult in the context of short telomeres develop pulmonary fibrosis. By using AAV9 vectors carrying the telomerase Tert gene to treat those mice, we explore the possibility of telomerase gene therapy as a possible treatment for IPF patients carrying short telomeres. To further understand gene expression changes undergoing in ATII cells upon telomerase activation, we isolated ATII cells from pulmonary...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by array Mus musculus Source Type: research
In conclusion, death is a natural part of human existence, but human progress is essentially a story of overcoming undesirable natural limits. In the near future, technological progress might make it possible to stop natural biological death. Should humankind embrace such technology? Yes: Even though such technology would not be without risks, the risks are almost certainly manageable. The benefits of ending natural death, on the other hand, are immense. Death is an obstacle that is slowing down human progress. If we remove that obstacle, humankind could increase the speed of both its moral and its epistemic progress. ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
In this study, we utilized H2O2- and X-ray-induced senescent human MSCs spiked in whole blood, as a model biological system, to demonstrate the rapid separation and analysis of senescent cells using our senescence chip. The optimized device was then used for an animal study to isolate senescent cells from the bone marrow of mice undergone total body irradiation (TBI) of X-ray. To achieve ultrahigh-throughput removal of senescent cells for blood purification, we enlarged the chip dimensions and stacked multiple chips to build a multiplexed system. We demonstrated that our scaled-up senescent chip could achieve a parallel pr...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Conditions:   Ischemic Heart Disease;   Acute Myocardial Infarction Interventions:   Biological: Stage 1-Low dose VM202;   Biological: Stage 1-Middle dose VM202;   Biological: Stage 1-High dose VM202;   Drug: Stage 2-Placebo;   Biological: Stage 2-Low dose VM202;   Biological: Stage 2-High dose VM202;   Device: C-Cathez® Catheter Sponsor:   ViroMed Co., Ltd. dba VM BioPharma Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Gene therapy: Autoimmune diabetes reversed in mice, Published online: 19 January 2018; doi:10.1038/nrendo.2018.4Gene therapy: Autoimmune diabetes reversed in mice
Source: Nature Reviews Endocrinology - Category: Endocrinology Authors: Source Type: research
We examined the transcriptional activation of the Klotho gene using three different systems: a Firefly luciferase (FLuc) and NanoLuc luciferase (NLuc) coincidence reporter system, a NLuc kn ock-in in Klotho 3′-UTR using CRISPR genomic editing, and two human cell lines: neuronal SY5Y cells and kidney HK-2 cells that express Klotho endogenously. The two sgRNAs enhanced Klotho expression at both the gene and protein levels. Our results show the feasibility of gene therapy for targeting Klotho using CRISPR technology. Enhancing Klotho levels has a therapeutic potential for increasing cognition and treating age-associated...
Source: Journal of Molecular Neuroscience - Category: Neuroscience Source Type: research
AAV-9 mediated phosphatase-1 inhibitor-1 overexpression improves cardiac contractility in unchallenged mice but is deleterious in pressure-overload, Published online: 19 January 2018; doi:10.1038/gt.2017.97AAV-9 mediated phosphatase-1 inhibitor-1 overexpression improves cardiac contractility in unchallenged mice but is deleterious in pressure-overload
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
CCAT1 stimulation of the symmetric division of NSCLC stem cells through activation of the Wnt signalling cascade, Published online: 19 January 2018; doi:10.1038/gt.2017.98CCAT1 stimulation of the symmetric division of NSCLC stem cells through activation of the Wnt signalling cascade
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Conditional replication of oncolytic viruses based on detection of oncogenic mRNA, Published online: 19 January 2018; doi:10.1038/gt.2017.99Conditional replication of oncolytic viruses based on detection of oncogenic mRNA
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Patients entered into our gene therapy study underwent complete physical and neurologic examinations before and after treatment, in addition to the visual studies. They also underwent many laboratory studies, which included hematology, coagulation, renal, and liver profiles. We did not detect any abnormalities other than visual loss. We had replied to this same query previously.1 Newman et  al2 described normal electrocardiographs, lumbar punctures, computed tomography scans, and magnetic resonance imaging in all 72 of their 11 778 patients.
Source: Ophthalmology - Category: Opthalmology Authors: Tags: Correspondence Source Type: research
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