Next-gen Sanfilippo Type A Gene Therapy May Move Into Clinical Trial

Next-gen Sanfilippo Type A Gene Therapy May Move Into Clinical Trial

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Next-gen Sanfilippo Type A Gene Therapy May Move Into Clinical Trial

Following the promising results of a second-generation gene therapy in a mouse model of Sanfilippo syndrome type A, a first trial in patients might be launched this year. The Sanfilippo Children’s Foundation reports that, if further work on this gene therapy continues to show potential, “there is hope to start a trial in 2021,” the foundation stated in a press release. Gene therapies are currently at the forefront of research for diseases with a genetic cause, including…

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