Stunning gene therapy breakthrough driven by great dedication and graft | Robin McKie

We need more than ever to celebrate advances in medical science – though they may take years to emergeThere has been a surprising outbreak of the use of the c-word among medical researchers over the past few days. Normally cautious in their language, they have nevertheless been wielding the term “cure” when discussing the long-term potential of two separate treatments for inherited ailments that were announced last week. Such enthusiasm is striking.In one case, scientists based at St Bartholomew ’s, London – who have been working on the inherited bleeding disorder haemophilia A –outlined how they had used a virus to carry the gene for the blood-clotting chemical, factor VIII (which patients lack) to their livers. Production of the missing chemical was restored and their bleeding halted. The development, according to theWorld Federation of Hemophilia, now points “the way to a cure” for the condition, which affects around 400,000 people worldwide.Continue reading...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Medical research Science Genetics Biology Cystic fibrosis Society Huntington's disease Alzheimer's Dementia Source Type: news

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Publication date: Available online 10 October 2020Source: Respiratory Medicine Case ReportsAuthor(s): Vipul Patel, Tilottama Majumdar, Isha Samreen, Harpreet Grewal, Thomas Kaleekal
Source: Respiratory Medicine Case Reports - Category: Respiratory Medicine Source Type: research
Authors: Zhao N, Xiang Q, Liu Z, Zhao X, Cui Y Abstract INTRODUCTION: There remains an unmet need for better anticoagulants. The phase I clinical trial is of great significance in the development of anticoagulants, and the design is special. This system review aims to provide insights for the design of future phase I clinical trials of anticoagulants. AREAS COVERED: We searched the database PubMed and ClinicalTrail.gov website, to collate the phase I clinical trial of anticoagulants in healthy people. The study protocol, inclusion exclusion criteria, safety and pharmacodynamic indexes were reviewed. EXPERT ...
Source: Expert Review of Clinical Pharmacology - Category: Drugs & Pharmacology Tags: Expert Rev Clin Pharmacol Source Type: research
DEMENTIA can be tricky to pick up on in the earliest stages. However, mounting research indicates a certain time of day when symptoms may be more noticeable. What time do you need to be on full alert?
Source: Daily Express - Health - Category: Consumer Health News Source Type: news
CONCLUSIONS: Individually customized, multicomponent exercise programs lead to improved levels of cognitive function, depression, and quality of life, especially among those who are more frail. PMID: 33029968 [PubMed]
Source: Journal of Clinical Neurology - Category: Neurology Tags: J Clin Neurol Source Type: research
Authors: Fentie K, Wakayo T, Gizaw G Abstract Background: Anemia defined as a low blood hemoglobin concentration is public health importance. The adolescence age group is the most neglected in public health and nutrition research as priorities are usually given to pregnant women, lactating mothers, and their children less than 2 years. Current Ethiopian Food and Nutrition policy included adolescent girls in the most at-risk group for nutritional demands; however, only a few published studies have assessed a deficiency of anemia and associated factors to tackle the intergenerational cycle of malnutrition. Object...
Source: Anemia - Category: Hematology Tags: Anemia Source Type: research
Authors: Gao Y, Xin L, Zhang YT, Guo XR, Meng QQ, Li ZS, Liao Z Abstract Background/Aims: Single-balloon enteroscopy (SBE) has been widely used in diagnosing small bowel disease. We conducted this study to systematically appraise its technical and clinical performance. Methods: Studies on SBE published by September 2018 were systematically searched. Technical and clinical performance data were collected and analyzed with descriptive or meta-analysis methods. Results: In total, 54 articles incorporating 4,592 patients (6,036 procedures) were included. Regarding technical parameters, the pooled insertion dept...
Source: Gut and Liver - Category: Gastroenterology Tags: Gut Liver Source Type: research
Publication date: Available online 9 October 2020Source: The Journal of Allergy and Clinical Immunology: In PracticeAuthor(s): Manuel Jorge Rial, Marcela Valverde, Victoria del Pozo, Francisco Javier González-Barcala, Carlos Martínez-Rivera, Xavier Muñoz, José María Olaguibel, Vicente Plaza, Elena Curto, Santiago Quirce, Pilar Barranco, Javier Domínguez-Ortega, Joaquin Mullol, César Picado, Antonio Valero, Irina Bobolea, Ebymar Arismendi, Paula Ribó, Joaquín Sastre
Source: The Journal of Allergy and Clinical Immunology: In Practice - Category: Allergy & Immunology Source Type: research
Conclusion: SLT induces relaxation of rat isolated tail artery through endothelium-independent mechanisms. The SLT-induced vasodilatation appeared to be jointly meditated by blockages of extracellular Ca2+ influx via receptor-gated and voltage-gated Ca2+ channels and inhibition of the release of Ca2+ from the sarcoplasmic reticulum. PMID: 33029174 [PubMed]
Source: Evidence-based Complementary and Alternative Medicine - Category: Complementary Medicine Tags: Evid Based Complement Alternat Med Source Type: research
Trump plans in-person rally on Monday as next presidential debate cancelled; Europe records 100,000 daily cases for first time; Canada at ‘tipping point’. Follow latest updatesEurope: Spain declares emergency in Madrid as Berlin emerges as hotspotUK workers to get two-thirds of wages if firms told to shutAustralia: Is Victoria ready to come out of lockdown?US presidential debate cancelled – live updatesSee all our coronavirus coverage5.42amBSTMy colleagueLuke Henriques-Gomes has this story giving you a summary of the main Covid-19 stories from Australia.A main point of the day has beenVictoria ’s pr...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Coronavirus outbreak Infectious diseases Science World news France Trump administration Donald Trump Source Type: news
Aβ-Induced Repressor Element 1-Silencing Transcription Factor (REST) Gene Delivery Suppresses Activation of Microglia-Like BV-2 Cells. Neural Plast. 2020;2020:8888871 Authors: Yu T, Quan H, Xu Y, Dou Y, Wang F, Lin Y, Qi X, Zhao Y, Liu X Abstract Compelling evidence from basic molecular biology has demonstrated the crucial role of microglia in the pathogenesis of Alzheimer's disease (AD). Microglia were believed to play a dual role in both promoting and inhibiting Alzheimer's disease progression. It is of great significance to regulate the function of microglia and make them develop in a favorabl...
Source: Neural Plasticity - Category: Neurology Authors: Tags: Neural Plast Source Type: research
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