Gene Therapies Make Compensatory Metabolic Adjustment More Precise, But That Still Isn't Damage Repair

Given a suitable delivery system, one that localizes to the desired target tissues to a far greater degree than to all other undesirable off-target tissues, the big advantage of a gene therapy is it precisely achieves the manipulation desired. It dials up or dials down expression for selected genes, alters the amount of proteins produced from those genes, and thereby changes cell behavior as a consequence - and that is all it does. One doesn't have the endless concern about off-target effects that characterize small molecule drug development. There are, of course, different challenges. Setting aside some adventurous technologies that won't be deployed in therapies any time soon, manipulating a few genes at a time is the present practical upper limit on gene therapy. Further, there is no viable delivery system for most target tissues in the body, if the goal is to maximize expression in a limited set of locations. Yes, a great many interesting technologies exist for use in animal studies, but the bounds of the possible are more limited when it comes to what is permitted in the clinic. Injecting a gene therapy vector into the bloodstream means that most of it will end up in the liver, lungs, and heart, and very little in lesser, smaller organs. Injecting a vector directly into tissue is prohibitively risky for most internal organs except in cases of very serious disease. Another pressing issue is that there is no proven gene therapy vector that can produce months ...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs

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Ovarian clear cell carcinoma (OCCC) and ovarian endometrioid carcinoma (OEC) are both associated with endometriosis but differ in histologic phenotype, biomarker profile, and survival. Our objectives were to refine immunohistochemical (IHC) panels that help distinguish the histotypes and reassess the prevalence of mismatch repair deficiency (MMRd) in immunohistochemically confirmed OCCC. We selected 8 candidate IHC markers to develop first-line and second-line panels in a training set of 344 OCCC/OEC cases. Interobserver reproducibility of histotype diagnosis was assessed in an independent testing cohort of 100 OCC/OEC ini...
Source: The American Journal of Surgical Pathology - Category: Pathology Tags: Original Articles Source Type: research
Discussion Anorexia nervosa is an eating disorder with an intense fear of being overweight (i.e. distorted body image), and self-starvation and excessive weight loss. It is more common in women (1.2%) than men (0.2%) but can be seen in both genders. Risk factors include high-intelligence, perfectionism/inflexibility, anxiety, activities where thinner body types are expected (e.g. dancer, diver, gymnast, long-distance runner, volleyball player, etc.) Other eating disorders include bulimia nervosa (i.e. cycles of binge eating and then purging), binge eating disorder (i.e. purging), orthorexia (i.e. obsessions with healthful ...
Source: - Category: Pediatrics Authors: Tags: Uncategorized Source Type: news
OBJECTIVES: To evaluate the efficacy of the simultaneous hypertonic saline solution and IV furosemide (HSS+Fx) for patients with fluid overload compared with IV furosemide alone (Fx). DATA SOURCES: Electronic databases (MEDLINE, EMBASE, CENTRAL, Cochrane Database of Systematic Reviews, PsycINFO, Scopus, and WOS) were searched from inception to March 2020. STUDY SELECTION: Randomized controlled trials on the use of HSS+Fx in adult patients with fluid overload versus Fx were included. DATA EXTRACTION: Data were collected on all-cause mortality, hospital length of stay, heart failure–related rea...
Source: Critical Care Medicine - Category: Emergency Medicine Tags: Online Review Article Source Type: research
The food that can reverse the damage done to blood vessels caused by a high-sodium diet.
Source: PsyBlog | Psychology Blog - Category: Psychiatry & Psychology Authors: Tags: Blood Pressure Source Type: blogs
Source: Acta Diabetologica - Category: Endocrinology Source Type: research
ConclusionsWe urgently need data on the effect of COVID-19 vaccination, in terms of maternal and fetal outcomes and vaccine related symptoms in high risk women during pregnancy and breastfeeding. It is important to run campaigns to promote vaccination, in particular in pregnant women at high risk to have severe COVID infection as those diabetics and/or obese.
Source: Acta Diabetologica - Category: Endocrinology Source Type: research
ConclusionsPNDM is a heterogenous disease with variable genotypes and clinical phenotypes among Egyptian patients.EIF2AK3, INS,ABCC8, and KCNJ11 mutations were the commonest causes of PNDM in the study cohort. All patients with KATP channel mutations were effectively treated with glyburide, reflecting the fact that genetic testing for patients with NDM is not only important for diagnosis but also for treatment plan and prognosis.
Source: Acta Diabetologica - Category: Endocrinology Source Type: research
This study confirmed that the PSI could be a quantitative index of vascular aging and has potential for use in inferring arterial stiffness with an advantage over the rAIx. A Profile of Michael Greve and the Segment of the Longevity Industry that He Supports Would that the popular media produced more popular science articles about the longevity industry like this one. It is not just a profile of someone trying to make a difference in the world by advancing the state of medici...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Using biochemical tools, scientists find that jellyfish are more than just floating bags of water
Source: Chemical and Engineering News - Category: Chemistry Authors: Source Type: research
In this study, we therefore analysed the influence of lithium treatment on lifespan and parameters of health during ageing in mice. To determine the concentration of lithium suitable to be administered in a longitudinal ageing study, we first tested the effects of lithium chloride (LiCl) in doses from 0.01 to 2.79 g LiCl per kg chow. C57Bl/6J mice fed with 1.05-2.79 g/kg LiCL in the diet showed lithium plasma levels between 0.4 and 0.8 mM/l. While plasma levels to 0.4 and 0.8 mM/l are well tolerated by human patients, at doses above 1.44 g LiCl/kg, we observed an obvious dose-dependent polydipsia combined with a dis...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
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