gene therapy Archive

gene therapyCreating real life superheroes

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Professor, author, and comic book enthusiast E. Paul Zehr looks at how near term technology could be used to create Marvel’s Captain America. Chasing Captain America explores what the real limits of being human are, how far we should bend …

canadaBridged nucleic acids in CRISPR RNAs improves CRISPR gene therapy accuracy by 10,000 times

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Canadian research (Alberta) shows that the use of bridged nucleic acids to guide Cas9 can improve its specificity by over 10,000 times in certain instances — a dramatic improvement. Basil Hubbard, Canada Research Chair in Molecular Therapeutics and an assistant …

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 …