Dr Changfa Guo, Professor Chunsheng Wang and their co-investigators from Zhongshan hospital Fudan University, Shanghai, China have established a novel hyperbranched poly(amidoamine) (hPAMAM) nanoparticle based hypoxia regulated ...
Jul 6, 2012
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The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie "Lorenzo's Oil."
Oct 4, 2017
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An experimental gene therapy essentially doubled the overall survival of patients with recurrent glioblastoma compared to the current standard of care, a researcher said Oct. 1 at the Cancer Therapy & Research Center (CTRC) ...
Oct 1, 2015
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An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
Jan 28, 2021
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A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of ...
Jan 30, 2024
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An article published in Experimental Biology and Medicine (Volume 242, Issue 13, July, 2017) reports that gene therapy may be used to as an intermediate therapy for newborns with surfactant protein deficiencies until lung ...
Jul 26, 2017
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Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. In new research presented today, scientists successfully used gene therapy to slow the progression ...
Nov 4, 2018
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Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the enzyme UGT1A1 which is responsible for metabolizing it into a substance that ...
Aug 17, 2023
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Their stories are divided into before and after. First, those long years of pain which flooded every moment—school, relationships, work.
Mar 18, 2024
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An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
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