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A clinical trial to test a gene therapy treatment for a rare liver disease

(AFM-T é l é thon) G é n é thon, a laboratory created by AFM-T é l é thon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease. The trial, sponsored by G é n é thon, will include 17 patients in four centres in Europe, and will assess safety and therapeutic efficacy of the gene therapy product developed by G é n é thon.
Source: EurekAlert! - Biology - Category: Biology Source Type: news

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In this study, we did not observe significant age-dependent upregulation of the prominent SASP cytokine Il6 in any tissue, although an upward trend was observed that was consistent in magnitude with previous observations in the heart and kidney. This modest age-related upward trend could be explained by a previous report which demonstrated that senescent cell-secreted IL-6 acts in an autocrine manner, reinforcing the senescent state, rather than inducing senescence or promoting dysfunction in neighboring cells. The decreased expression of Il6 with age we observed in the hypothalamus could be indicative of a lack or ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
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Source: Cell Transplantation - Category: Cytology Authors: Tags: Cell Transplant 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
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Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Discussion of advocacy for the cause is a usual feature of our community, as we try things and attempt to make progress in persuading the world that rejuvenation research is plausible, practical, and necessary. There are more people engaged in advocacy now than at any time in the past decade, and so discussions of strategy come up often. New ventures kicked off in 2017 include the Geroscience online magazine, and among the existing ventures the LEAF / Lifespan.io volunteers seem to be hitting their stride. The mainstream media continues to be as much a hindrance as a help, and where it is a help you will usually find Aubre...
Source: Fight Aging! - Category: Research Authors: Tags: Of Interest Source Type: blogs
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Source: Journal of the American College of Surgeons - Category: Surgery Authors: Tags: Scientific forum Source Type: research
Abstract Crigler‐Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe neonatal unconjugated hyperbilirubinemia with a lifelong risk of neurological damage and death. Liver transplantation is the only curative option, which has several limitations and risks. We applied an in vivo gene targeting approach based on the insertion, without the use of nucleases, of a promoterless therapeutic cDNA into the albumin locus of a mouse model reproducing all major features of CNSI. Neonatal transduction with the donor vector resulted in the complete rescue from neonatal lethality, with a therapeutic r...
Source: EMBO Molecular Medicine - Category: Molecular Biology Authors: Tags: Report Source Type: research
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Source: Acta Pharmaceutica Sinica B - Category: Cancer & Oncology Source Type: research
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Authors: Zhao X, Voutila J, Ghobrial S, Habib NA, Reebye V Abstract The prognosis for hepatocellular carcinoma (HCC) remains poor and has not improved in over two decades. Most patients with advanced HCC who are not eligible for surgery have limited treatment options due to poor liver function or large, unresectable tumors. Although sorafenib is the standard-of-care treatment for these patients, only a small number respond. For the remaining, the outlook remains bleak. A better approach to target "undruggable" molecular pathways that reverse HCC is therefore urgently needed. Small activating RNAs (saRNAs)...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
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