干细胞领域知识发现平台

[2019-09-09] First gene therapy for β-thalassemia approved

简介: Bluebird Bio has been granted the go-ahead to market its gene therapy for the blood disorder β-thalassemia. Zynteglo gained conditional market approval from the European Commission in June to treat transfusion-dependent β-thalassemia in patients 12 years and older who have no other treatment options.In people with the inherited disorder β-thalassemia, the oxygen-transport protein hemoglobin in red blood cells is defective.Credit: nobeastsofierce Science / Alamy Stock Photo.The therapy adds a cor

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[2019-09-06] Gene therapy boom continues

简介: In September 1999, gene therapy research ground to a halt with the tragic death of Jesse Gelsinger. Twenty years on, large pharmaceutical firms are fiercely vying for new gene therapy candidates and manufacturing capabilities.In August, for instance, Pfizer invested US$500 million into the construction of a gene therapy manufacturing site. The move follows Pfizer’s $600 million acquisition of Bamboo Therapeutics, a biotech with an adeno-associated virus (AAV) gene therapy platform, in 2016.Pfize

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[2019-09-04] Baby elephants, painkiller prescriptions and Russian radiation clues

简介: POLICYBan on exporting baby elephants Tough new trade restrictions for dozens of wildlife species, including elephants, have been agreed at a meeting of parties to the Convention on International Trade in Endangered Species (CITES) in Geneva, Switzerland, which ended on 28 August. Nations voted to ban the practice of taking elephant calves from the wild and selling them to zoos overseas, and to allow the export of wild elephants only in exceptional circumstances. Countries also agreed to regulat

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[2019-09-03] Daily briefing: ESA satellite forced to move to avoid SpaceX collision

简介: Hello Nature readers, would you like to get this Briefing in your inbox free every day? Sign up here.The transparent cornea protects the eye from damage.Credit: Ralph C. Eagle Jnr/Science Photo LibraryA Japanese woman in her forties has become the first person in the world to have her cornea repaired using corneal cells made from induced pluripotent stem (iPS) cells. iPS cells are made by reprogramming adult skin cells from a donor into an embryonic-like state, from which they can transform into

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[2019-09-03] The neurobiological mechanisms behind schizophrenia may depend on gender

简介: The neurobiological pathophysiology of schizophrenia differs significantly between males and females, according to a new study. The findings suggest a possible need for more sex-specific treatments for schizophrenia. The study was the first to identify a number of sex-specific genes related to schizophrenia using neurons derived from induced pluripotent stem cells. The results were published in Nature Communications.Co-ordinated by the University of Eastern Finland, the University of Helsinki an

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[2019-09-02] Woman is first to receive cornea made from ‘reprogrammed’ stem cells

简介: The transparent cornea protects the eye from damage.Credit: Ralph C. Eagle Jnr/Science Photo LibraryA Japanese woman in her forties has become the first person in the world to have her cornea repaired using reprogrammed stem cells.At a press conference on 29 August, ophthalmologist Kohji Nishida from Osaka University, Japan, said the woman has a disease in which the stem cells that repair the cornea, a transparent layer that covers and protects the eye, are lost. The condition makes vision blurr

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[2019-09-02] Enzyme known for promoting cancer found to also protect healthy cells

简介: New research from the University of Maryland and the National Institutes of Health reveals a new role for the enzyme telomerase. Telomerase's only known role in normal tissue was to protect certain cells that divide regularly, such as embryonic cells, sperm cells, adult stem cells and immune cells. Scientists thought telomerase was turned off in all other cells, except in cancerous tumors where it promotes unlimited cell division.The new study found that telomerase reactivates in normal adult ce

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[2019-08-30] PastCast: Monoclonal antibodies, from basic science to blockbuster drugs

简介: Kerri SmithThis is the Nature PastCast, each month raiding Nature’s archive and looking at key moments in science. In this show, the beginnings of some blockbusters in the 1970s.MusicVoice of Nature: John HoweNature, August 7, 1975.MusicLara MarksFrom the late nineteenth century, scientists began to wonder whether antibodies could be the next magic bullet in medicine.Greg WinterI had no idea that it was going to have the impact that it has now. At that point, I don’t think anyone had realised th

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[2019-08-29] Pancreas on a chip: Scientists combine organ-on-a-chip and stem-cell technologies

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[2019-08-28] Signal blocks stem cell division in geriatric brain

简介: Scientists from Basel have investigated the activity of stem cells in the brain of mice and discovered a key mechanism that controls cell proliferation. According to the researchers, the gene regulator Id4 controls whether stem cells remain in a state of rest or enter cell division. The results were published in Cell Reports and may be relevant for treating neurodegenerative disease in human brains.Whether stem cells also occur in the human brain has long been controversial. Today, it is conside

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