Phase 1/2 trial of gene therapy treatment for X-linked retinitis pigmentosa begins – Healio
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Phase 1/2 trial of gene therapy treatment for X-linked retinitis pigmentosa begins
Healio The gene therapy, AAV-XLRPGR, uses adeno-associated virus to deliver a codon-optimized copy of the retinitis pigmentosa GTPase regulator gene into the cells of the eye, the release said. “The current trial is a multicenter open-label, dose-escalation … |
Genetic Literacy Project |
No longer waiting idly: Parents of patients are establishing gene therapy programs
Genetic Literacy Project In recent years, gene therapies have become safer and better at hitting their intended targets in the body, leading to a handful of remarkable cures in clinical trials. Advocates for rare-disease patients—especially determined parents—are … |
CNET |
Gene therapy: What personalized medicine means for you
CNET Thuy Truong thought her aching back was just a pulled muscle from working out. But then came a high fever that wouldn’t go away during a visit to Vietnam. When a friend insisted Truong, 30, go to an emergency room, doctors told her the last thing she … |
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AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
Yahoo Finance AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s … |
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Dr. Carl H. June, Cancer Immunotherapy Pioneer, Joins CytoSorbents Scientific Advisory Board
Yahoo Finance MONMOUTH JUNCTION, N.J., March 22, 2017 /PRNewswire/ — CytoSorbents Corporation (CTSO), a critical care immunotherapy leader commercializing its flagship CytoSorb® blood filter to treat deadly inflammation in critically-ill and cardiac surgery … |
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Dr. Carl H. June, Cancer Immunotherapy Pioneer, Joins CytoSorbents Scientific Advisory Board
PR Newswire (press release) MONMOUTH JUNCTION, N.J., March 22, 2017 /PRNewswire/ — CytoSorbents Corporation (NASDAQ: CTSO), a critical care immunotherapy leader commercializing its flagship CytoSorb® blood filter to treat deadly inflammation in critically-ill and cardiac … |