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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AbstractAdenovirus 5 (Ad-5) infection is a common cause of acute respiratory infections and the main vector used in gene therapy. There are few studies on the relationship of Ad-5 to obesity. In the present study, we evaluated the chronic effects of Ad-5 infection on golden (Syrian) hamsters fed either a balanced diet (BD) or a high-fat diet (HFD). After a single inoculation with Ad-5 (1 × 107 pfu), the body weight of the animals was measured weekly. Medium-term (22 weeks) serum biochemical analyses and long-term (44 weeks) liver morphology, adiposity, and locomotive functionality (movement velocity) assessments were...
Source: Archives of Virology - Category: Virology 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
CONCLUSION: Our results show that gene therapy by delivering GS is a promising alternative for treatment of hyperammonemia in acute-on-chronic liver failure patients with HE. PMID: 30600292 [PubMed - in process]
Source: Annals of Hepatology - Category: Gastroenterology Tags: Ann Hepatol Source Type: research
In conclusion, there are many anti-aging strategies in development, some of which have shown considerable promise for slowing down aging or delaying the onset of age-related diseases. From multiple pre-clinical studies, it appears that upregulation of autophagy through autophagy enhancers, elimination of senescent cells using senolytics, transfusion of plasma from young blood, neurogenesis and BDNF enhancement through specific drugs are promising approaches to sustain normal health during aging and also to postpone age-related diseases. However, these approaches will require critical assessment in clinical trials to determ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Researchers recently demonstrated that they could rescue a form of mitochondrial dysfunction in mice by importing a gene from a sea squirt species. This is particularly interesting in the context of aging, as it appears to be possible to use this approach to work around any sort of damage to complexes III and IV in the mitochondrial electron transport chain (ETC). Every cell is equipped with a herd of mitochondria that act as generators, packaging the chemical energy store molecules used to power the cell. The ETC is central to this function. The protein complexes that make up the ETC are made up of a mix of protein...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
This study is the first to demonstrate long-term safety and efficacy of hepatocyte-directed gene therapy in a large animal model. We conclude that hepatocyte-directed ex-vivo gene therapy is a rational choice for further exploration as an alternative therapeutic approach to whole organ transplantation for metabolic liver disease, including HT1. PMID: 30477316 [PubMed - as supplied by publisher]
Source: Cell Transplantation - Category: Cytology Authors: Tags: Cell Transplant Source Type: research
BackgroundAdvances in red blood cell (RBC) transfusion and chelation have improved the prognosis of patients with transfusion-dependent β-thalassemia (TDT); however, many patients experience organ damage due to iron overload and other complications. While potentially curative, allogeneic hematopoietic stem cell transplantation confers significant risks of morbidity and mortality and is limited by donor availability. Gene therapy (GT) has the potential to be an effective treatment option for patients with TDT without some of these limitations. LentiGlobin GT contains autologous CD34+ cells transduced ex vivo with the B...
Source: Blood - Category: Hematology Authors: Tags: 112. Thalassemia and Globin Gene Regulation: Clinical Source Type: research
Hemophilia B (HB) is a prime model for gene therapy. While data from current clinical trials using AAV-mediated liver-targeted FIX gene therapy are very encouraging, this protocol can only be applied to adults without liver disease or anti-AAV antibodies. Thus, developing another gene therapy protocol is desired. Our previous studies have demonstrated that platelet-targeted FIX expression driven by the platelet-specific αIIb promoter (2bF9) restores hemostasis and induces immune tolerance in HB mice (Chen et al. Mol Ther 2014). To improve the efficacy, we used a codon-optimized hyperfunctional FIX Padua (2bCoF9R338L)...
Source: Blood - Category: Hematology Authors: Tags: 321. Blood Coagulation and Fibrinolytic Factors: Animal Models and Therapeutic Targets in Thrombosis and Hemostasis Source Type: research
BackgroundSickle cell disease (SCD) is a progressively debilitating genetic disease causing significant morbidity and early mortality for which a universal curative therapy is lacking. Expression of an anti-sickling β-globin via gene transfer into hematopoietic stem cells (HSCs) may reduce or eliminate SCD symptoms. LentiGlobin Drug Product (DP) contains autologous CD34+ cells transduced with the BB305 lentiviral vector (LVV) encoding β-globin with an anti-sickling substitution (T87Q). The safety and efficacy of LentiGlobin in adults with severe SCD is being evaluated in the ongoing multi-center Phase 1 study HGB...
Source: Blood - Category: Hematology Authors: Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research
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
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