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Gene therapy to treat rare bleeding disorder - 0 views

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    The European Union's health regulator on Friday said it had recommended granting a conditional marketing authorisation for a gene therapy by Australian drugmaker CSL Ltd and partner uniQure N.V. , to treat haemophilia B, a rare bleeding disorder which is caused by genetic anomalies. About one in 40,000 people are affected by the inherited disorder, caused by a gene mutation that hampers the body's ability to make clotting protein factor IX. If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said. CSL had acquired exclusive global rights to Hemgenix in May last year from uniQure N.V.
pharmacybiz

Novo Nordisk's bet on gene-editing technology in pharma - 0 views

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    Capitalising on strong demand for its obesity therapies, Novo Nordisk's growing appetite for deals has fuelled a bet on a U.S. gene-editing company called Life Edit Therapeutics. The Danish drugmaker's collaboration with Durham, North Carolina-based Life Edit Therapeutics - owned by ElevateBio, a cell and gene therapy company in Waltham - is focused on up to seven programs for rare genetic disorders as well as cardiometabolic diseases. At the heart of the tie-up is Life Edit's technology, called base editing, which is designed to make precise changes to the human genome by tweaking one base - or letter - into a different one without affecting other letters. Single-letter mistakes, called point mutations, can give rise to genetic diseases.
vinita choudary

'Stem cell gene linked to cancer' - 0 views

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    Scientists have found a mutation in stem cell genes, which they claim could be linked to development of leukaemia and other cancers. An international team, led by Peter Papathanasiou of The Australian National University, has completed a three-year screening project to find the genes that control development and turnover of stem cells. Stem cells can grow into any other kind of cell in the body, making them a hot area of research to find new treatments for diseases and injuries
pharmacybiz

NICE recommends Tepotinib for advanced lung cancer - 0 views

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    'Tepotinib' has been recommended by the National Institute for Health and Care Excellence (NICE) to patients with a specific gene mutation of advanced non-small-cell lung cancer (NSCLC). The new potentially life-extending drug is available from Thursday (14 April). It has been recommended for routine use across the NHS in England through Project Orbis, a programme to review and approve promising cancer drugs helping patients access treatments faster. NICE has published its final appraisal document recommending tepotinib (also known as Tepmetko and produced by company Merck Serono Ltd) as an option for treating advanced NSCLC with METex14 skipping gene alterations in adults. People with METex14 skipping alterations of NSCLC make up between 1-2% of all adults with lung cancer in England. Tepotinib, which requires people to take two tablets once daily, provides a new targeted treatment for adults with METex14 skipping gene alterations. Just over 700 people in England would be eligible to receive tepotinib as either a first or second-line treatment.
pharmacybiz

MHRA : Approved Casgevy for Sickle Cell & β-thalassemia - 0 views

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    The Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised an innovative and first-of-its-kind gene-editing treatment for sickle-cell disease and transfusion-dependent β-thalassemia. Known as Casgevy (exagamglogene autotemcel), the treatment is based on the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. Casgevy is the first medicine to be licensed that uses CRISPR, and it is to be used for treating patients aged 12 and over. To date, a bone marrow transplant has been the only permanent treatment option for these life-long conditions, which in some cases can be fatal. The MHRA's authorisation came after a rigorous assessment of its safety, quality and effectiveness.
pharmacybiz

Life-Saving Treatment For Rare Disease Affecting Babies:NHS - 0 views

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    The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.
Terry Hess

TALENs Technology for Targeted Genome Editing - 0 views

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    Creative Animodel's scientists are researching over gene editing methods and developed TALENs technology to use for gene editing. TALENs are a process of binding proteins in DNA sequence. It's a very powerful approach for genome editing in most of the animal models.
Terry Hess

Pushing gene functionality study capabilities with transgenic animal models - 0 views

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    Transgenic animal models make the study of biological and chemical compounds intended for human use safer and more efficient. The technology also makes it possible to study the gene function and regulation of the organism
Terry Hess

Brief Introduction of Transgenic Animal Model - 0 views

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    A transgene is an artificial gene whose structure contains all the critical elements for gene expression. To create a transgene, recombinant DNA (rDNA) is needed. The process for making rDNA is a "cut and paste" method requiring the use of restriction enzymes and DNA ligase.
Terry Hess

RNA Interference for Gene Knockdown and Silence - 0 views

RNA interference (RNAi) is the process by which the expression of a target gene can be effectively silenced or knocked down.http://tinyurl.com/kpu6odw

RNAi gene knockdown services RNAi-mediated

started by Terry Hess on 29 Jan 15 no follow-up yet
insightscare

A slight genetic rearrangement can save lives from Malaria - Insights care - 0 views

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    A revolutionary discovery by the researchers have proved that the most severe form of malaria, caused by the parasite Plasmodium falciparum,is linked with natural variation in human red blood cell genes.
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    A revolutionary discovery by the researchers have proved that the most severe form of malaria, caused by the parasite Plasmodium falciparum,is linked with natural variation in human red blood cell genes.
levinson AJ

What are the advantages from Reparagen? - 0 views

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    Supports a normal, healthy Inflammatory Response. Reactivates the joint health gene IGF-1 in Human Chondrocytes, Supports normal cartilage synthesis.
Gene Support

GeneSupport - Gene Based Weight Loss Diet Plan in Pune - 0 views

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    Weight loss diet plan is not the same for everybody, as individual's metabolism is different. Therefore, the diet plan should be different for different individual. GeneSupport has introduced first DNA test for weight loss in Pune, India, offering personalized diet plans and exercise advice by sports medicine and fitness expert. Wherein, continual followup is taken of the diet plan suggested, of how your body responses and improve one's health. For more information about the test please visit the dedicated DNA weight loss test at http://genesupport.in
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    Weight loss diet plan is not the same for everybody, as individual's metabolism is different. Therefore, the diet plan should be different for different individual. GeneSupport has introduced first DNA test for weight loss in Pune, India, offering personalized diet plans and exercise advice by sports medicine and fitness expert. Wherein, continual followup is taken of the diet plan suggested, of how your body responses and improve one's health. For more information about the test please visit the dedicated DNA weight loss test at http://genesupport.in
antony dj

Herbalife Products Reviews - 0 views

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    Herbalife Products Reviews : The easiest way to Count in With Subaltern Fat Genetics does witticism a share in avoirdupois, of education, but not as immense a role as you do. Most health gurus say the intention of genes forcing you to be impenetrable is a parable. For the number of us, genes may set the inferior boundaries of our coefficient, but we set the higher boundaries by our content selections. Withal , everyone knows that more of us change a way to put on metric as we get senior. Additionally, if there's one attribute we won't preclude, it's the ageing affect. But we present be fit to quit consumption solon and effort little as we get sr.. And prototypical off, you've got to utilise.
antony dj

Best Herbalife Diet Review - 0 views

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    Best Herbalife Diet Review : The easiest way to Matter in With Lowly Fat Genetics does music a tune in avoirdupois, of way, but not as vast a enactment as you do. Most eudaimonia gurus say the line of genes forcing you to be full is a apologue. For the age of us, genes may set the move boundaries of our unit, but we set the higher boundaries by our substance selections. Withal , everyone knows that numerous of us make a disposition to put on coefficient as we farm older. Additionally, if there's one statement we won't preclude, it's the old transmute. But we leave be fit to cease uptake much and exertion little as we get senior. And firstly off, you've got to energy.
Angelina Princess

Effects of Genetic Markers on Testosterone Level - 0 views

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    A study was carried out in which genomes of more than 1400 men were examined. Changes were found with the genes that had low levels of testosterone levels in the blood. These changes were named as "risk markers." Men having three or more of these risk markers have chances of 6.5 times low testosterone level than in men having no risk markers. Genetic markers have strong effect on testosterone levels along with some other factors that cause low testosterone levels in men; it includes smoking, age, and body weight.
Terry Hess

Conditional Knockout Model Service Provider - 0 views

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    Creative Animodel is rolling out a tissue particular conditional knockout scientific type of mouse in which a focus on gene could be specially deletion from the sole body organ. In order to carry out that Creative Animodel employ Tetracycline-Inducible models in addition to Cre/lox System.
fnfdoc

Stroke Genetics And Risk Factors | Your Health Our Priority - 0 views

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    Stroke is a cerebrovascular syndrome that involves the blockage and bursting of blood vessels in the brain. It shows a multifactorial pattern of inheritance. High cholesterol levels, high blood pressure, hypertension and obesity are the key causes of stroke besides inherited genes.
pharmacybiz

GSK And Arrowhead To Develop drug For Fatty Liver Disease - 0 views

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    Arrowhead Pharmaceuticals on Monday (November 22) entered a drug development deal with GlaxoSmithKline (GSK) under which the British drugmaker will develop and market Arrowhead's potential treatment for patients with fatty liver disease NASH. Under the pact, Arrowhead said it would get an upfront payment of $120 million and is eligible for additional milestone payments including up to $190 million at first commercial sale of the product, and up to $590 million in sales-related milestone payments. The drug candidate, ARO-HSD, is currently in an early-to-mid stage trial for nonalcoholic steatohepatitis (NASH), a fatty liver disease. It is based on RNA interference technology, where genes that contribute to disease are silenced.
pharmacybiz

Retinoblastoma :NHS rolled out test to treat babies - 0 views

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    NHS England is set to roll out a new test this week that will help doctors to spot a rare form of eye cancer in babies in the womb. The NHS test developed at Birmingham Women's and Children's NHS Foundation Trust, identifies the risk of developing retinoblastoma in babies that can be monitored and treated sooner - increasing the chance of saving their eyesight and potentially their lives. The new non-invasive test can detect changes in the genes in DNA and is likely to identify around 50 infants with retinoblastoma each year, in the latest example of the NHS harnessing the power of genomics to diagnose and treat patients faster and more effectively. Non-Invasive Prenatal Diagnosis (NIPD) also means parents can be informed early in pregnancy if their child is at risk. "The blood sample test is taken from the mother before birth and tested and analysed for mutations, which can determine with almost 100 per cent accuracy if the baby will develop retinoblastoma," the NHS said.
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