gene therapy Archive

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 …

antiagingSupercentenarian may have genes that protect against bad effects of aging

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James Clement has collected blood, skin or saliva from supercentenarians in 14 states and seven countries over a six-year period. Lifestyle and luck, it seems, still factor heavily into why people live into their 90s and 100s. However, supercentenarians are …

gene therapyJapanese researchers make chickens that lay egss with low cost cancer drugs

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The National Institute of Advanced Industrial Science and Technology (AIST) Kansai in Osaka Prefecture has succeeded in making hens lay eggs that contain a pharmaceutical agent that can be used to treat such diseases as cancer and hepatitis, it has …

cancerFDA approves $475K Leukemia gene therapy treatment with 83% success rate

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The Food and Drug Administration on Wednesday announced what the agency calls a “historic action” — the first approval of a cell-based gene therapy in the United States. The FDA approved Kymriah, which scientists refer to as a “living drug” …