gene-therapy
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Updates from uniQure about their gene therapy for Huntington's disease
uniQure is conducting safety trials of the first gene therapy for HD. A recent press release provided a 1 year update on the first group of 10 people who underwent surgery to receive this experimental drug. Let’s talk more about what it means.
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A first for CRISPR gene editing could have wider applications for human disease
A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?
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Good news from uniQure: gene therapy trial on track, and promising data in animals
The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
By Dr Leora Fox -
Caution urged for the use of gene-editing technology CRISPR
A recent series of studies on the gene-editing method CRISPR have raised concerns about the suitability of this technology for the treatment of genetic illnesses such as Huntington's disease
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Exciting new Huntingtin lowering tool described
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
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New roles for huntingtin: removing a healthy protein to understand its function
Completely removing normal huntingtin in adults may disrupt healthy brain function, a recent study suggests.
By Dr Leora Fox