gene-therapy
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Unpacking recent gene therapy press
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.
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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 uniQureās 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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Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants āInvestigational New Drugā status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntingtonās Disease patients
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Advances on many fronts in the battle against the protein that causes Huntington's disease
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
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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 -
Important advances in next generation genome editing tools for Huntington's Disease
Work with genome editing techniques (zinc fingers and CRISPR) brings these tools closer to use in HD clinical trials
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Could a new "jaw-dropping" breakthrough help treat Huntington's Disease?
The news has recently been full of stories about CRISPR, a new "jaw-dropping" DNA-editing technology. Hype or hope?