A hospital nurse for 33 years, Nanette Miller would call her husband Frank at the end of each shift to let him know she was coming home. On Dec. 7, 2018, the phone call came with a somber declaration: "I can't do this anymore."
Aug 6, 2020
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The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 2, 2020
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Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with ...
Jun 15, 2020
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An initial trial in patients indicates that a new genetic treatment for complete color blindness, developed by research groups based in Tübingen and Munich is safe. Preliminary evidence for its efficacy has also been obtained.
May 1, 2020
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A new study led by the University of Bristol has shown a common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality ...
Apr 21, 2020
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Researchers at the University of Cincinnati have uncovered a potentially more beneficial treatment regimen for patients with metastatic melanoma.
Feb 26, 2020
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A pilot gene therapy treatment for sickle cell disease, restoring patients' ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children's Hospital ...
Jan 10, 2020
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For the first time in the United States, a gene editing tool has been used to treat advanced cancer in three patients and showed promising early results in a pilot phase 1 clinical trial. So far the treatment appears safe, ...
Nov 19, 2019
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Researchers at UC Santa Cruz have demonstrated a new, rapid method to obtain donor stem cells for bone marrow transplants using a combination of Viagra and a second drug called Plerixafor.
Oct 10, 2019
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At the Association for Glycogen Storage Disease's 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children's presented his groundbreaking, one-year clinical trial results for the novel ...
Sep 20, 2019
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