Chasing Genzyme, Cambridge biotech AvroBio lifts $60M to fund Fabry illness gene medication experience

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Fresh animal research lifts worries about high-dose gene medication

Science photo Co/Science SourceNew animal research lifts worries about high-dose gene therapyA gene medication experience that soon led to dramatic advantages for babies born by a fatal neuromuscular condition has lifted wishes for Utilizing a similar approach to treat other illnesses. Based on these latest animal results, Wilson is urging investigators planning to treat patients by high-dose AAV9 gene medication to look for similar side influences. Despite that caution, he & others in the gene medication field tell that does not mean ongoing clinical trials by the gene carrier ought be halted. "This is a large bargain, potentially," tells Terence Flotte, a gene medication investigator at the University of Massachusetts School of Medicine in Worcester & editor-in-chief of Human Gene medication , that published the Fresh research on-line on 29 January. In their commentary, Flotte & a co-author write that AAV9 gene medication trials ought still because they are potentially keeping lives.

New animal study raises concerns about high-dose gene therapy

Alarm sounds for gene medication toxicity, however was the worry legitimate?

according to [+]Enlarge A cartoon illustrating the structure of AAV9, a virus used to deliver gene medication into the nervous system. 2012, DOI: ten.1128/JVI.07232-eleven)A controversial Fresh research with veteran gene medication scholar James Wilson at the University of Pennsylvania is rousing worries about the security of high-dose gene medication trials. Many gene medication stocks dropped on the break news. Wilson was the lead scholar behind a highly publicized gene medication experience that resulted in the dying of 18-year-old Jesse Gelsinger in 1999. such as many other gene medication companies, Solid Biosciences is Utilizing a particular kind of AAV, called AAV9, that easy gets in the nervous system.




collected by :Lucy William
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