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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Adeno-associated virus (AAV) vectors have become one of the most widely used systems for gene delivery. These vectors have been tested in a diverse range of pre-clinical models, ranging from liver disease to heart failure, with highly promising results. In fact, there were over 100 abstracts presented at the American Society of Gene and Cell Therapy (ASGCT) 21st Annual Meeting in Chicago in 2018 that described the use of AAV vectors in pre-clinical or clinical studies. A quick review of selected categories related to gene delivery revealed that AAV comprised nearly half of the abstracts describing the use of viral or non-v...
Source: Heart, Lung and Circulation - Category: Cardiology Authors: Tags: Editorial 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
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
Startup factory Third Rock Ventures is launching a three-pronged attack on liver diseases with $140 million and an unusually early and large assist from a Japanese drug maker. Ambys Medicines Inc. will get $100 million from Takeda Pharmaceutical Co. Ltd. — including $80 million in research and development commitments — on top of $40 million from Third Rock to pursue cell therapy, gene therapy and traditional small-molecule approaches to restoring and replacing live r function lost in a variety…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
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
Autologous hepatocyte transplantation after ex vivo gene therapy is an alternative to liver transplantation for metabolic liver disease. Here we evaluate ex vivo gene therapy followed by transplantation of single-cell or spheroid hepatocytes.
Source: Surgery - Category: Surgery Authors: 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
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
In this study, we attempted to show the effectiveness of human iPS cell-based liver-like tissue engineering at an extrahepatic site for treatment of hemophilia B, also called factor IX (FIX) deficiency. HLCs were transplanted under the kidney capsule where the transplanted cells could be efficiently engrafted. Ten weeks after the transplantation, human albumin (253 μg/mL) and α-1 antitrypsin (1.2 μg/mL) could be detected in the serum of transplanted mice. HLCs were transplanted under the kidney capsule of FIX-deficient mice. The clotting activities in the transplanted mice were approximately 5% of those in wild...
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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