Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that delivers therapeutic genes—for use in gene therapy for sickle cell disease. In advanced lab tests ...
Oct 2, 2019
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After over a decade of preclinical research and development, a new gene therapy treatment for Sickle Cell Anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged ...
Dec 4, 2018
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Many research groups have recently explored human adaptation and successfully identified candidate genes to high altitude living among three major far-flung global populations: Tibetans, Ethiopians and Peruvians.
Sep 21, 2017
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If you recognise the words "CRISPR-mediated gene editing", then you'll know that our ability to alter DNA has recently become much more efficient, faster and cheaper.
Feb 16, 2017
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A new study in JAMA provides evidence that hemoglobin A1c (HbA1c), a common blood biomarker used to measure blood sugar over time, may not perform as accurately among African-Americans with sickle cell trait and could be ...
Feb 7, 2017
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The ability to precisely edit DNA via CRISPR technology has emerged as the one of the most powerful advances in biology. A new paper showing repair of a genetic mutation in human blood cells represents an important step towards ...
Oct 19, 2016
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A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's ...
Sep 6, 2016
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For the last several decades, scientists worldwide have been seeking to harness the power of stem cells to develop therapies for human diseases and conditions. At UCLA's Broad Stem Cell Research Center, the potential to bring ...
Jun 14, 2016
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As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist-oncologist ...
May 26, 2016
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Tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering.
Jan 12, 2016
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