gene therapy Archive

crisprGene therapy treatment for blood disease approved in Europe

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Genetically modification of humans has been been approved for the first time in Europe. Swiss-based biotech company Crispr Therapeutics hope to find a way of curing blood disease beta thalassaemia. China has approved similar gene therapy trials. A trial into …

alzheimer'sGene APOE4 causes Alzheimers disease in humans and a fix has been created

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Researchers have found differences in how Alzheimers disease originates and develops in mice and humans. They determined which gene causes Alzheimers in humans and have developed a fix for it. Dementia and Alzheimer’s disease aren’t the same. Dementia is an …

diseaseGene therapy for common blood disorders

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In a landmark study that could lead to new therapies for sickle cell anaemia and other blood disorders, UNSW Sydney-led researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells to boost their production of foetal …

chinaChina leads world with 8 CRISPR trials to treat cancer and CRISPR medicine is a national priority

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China has at least eight CRISPR gene therapy clinical trials to treat various forms of cancer. CRISPR is being tested as a treatment for cancers of the lung, bladder, cervix and prostate. Clinicaltrials.gov lists all of the clinical trials in …

gene therapyGene therapy getting approved to treat major diseases of muscle and blood

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Researchers review the pioneering work that led the gene therapy field to its current state, describe gene-editing technologies that are expected to play a major role in the field’s future, and discuss practical challenges in getting these therapies to patients …

gene therapyBeyond Crispr -Cas9 Gene editing

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Crispr/Cas9 is a riboprotein complex composed of a short strand of RNA and an efficient DNA-cutting enzyme. Crispr/Cas9 sometimes cuts in the wrong places. And it has no off-switch. Epigenetic Editing Other proteins—like ones that activate gene expression—can be combined …

gene therapyCRISPR gene therapy preserves hearing

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Researchers deliver CRISPR-Cas9 directly into the inner ear hair cells of mice, preventing hearing loss in animal model of genetic progressive deafness. Above – Credit : Gao et al./Nature 2017. Confocal microscopy images of mouse cochlea; hair cells in green. …

crisprCRISPR extends lifespan of Lou Gehrigs disease mice by 25%

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University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig’s disease, in mice, extending their lifespan by 25 percent. The therapy delayed the …

gene therapyTHOR gene could be critical to curing cancer

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Researchers have identified and characterized a new type of RNA protein called THOR that plays a role in cancer development — and could serve as a target for drug development. Researchers at the University of Michigan Comprehensive Cancer Center uncovered …

gene therapyGene therapy to fix failing hearts works in pigs and will be tried in humans in 2018

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Scientists have been able to reverse heart failure in pigs by delivering a new gene to the heart that makes it better at pumping blood and supplying the body with oxygen. The goal is to reverse heart failure with a …