Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons ...
Feb 19, 2021
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Researchers at Children's Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently ...
Jan 29, 2021
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Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment options. A team led by investigators at Massachusetts ...
Jan 9, 2021
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A new review of research bringing together patient, carer and public views of cell and gene therapies has highlighted a need for appropriate education to better inform people including how clinical trials work and the risks ...
Dec 8, 2020
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For Evie Junior, living with sickle cell disease has been like running a marathon.
Nov 30, 2020
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Is the eye a window to the brain in Sanfilippo syndrome, an untreatable form of childhood-onset dementia, Australian researchers ask in a new publication.
Nov 30, 2020
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A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said Thursday.
Oct 15, 2020
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The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible ...
Oct 9, 2020
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A new publication by scientists from Children's Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the ...
Sep 9, 2020
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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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