Articles with the topic: gene-therapy

A first for CRISPR gene editing could have wider applications for human disease

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?

Daniel O’ReillyJuly 21, 2021

Good news from uniQure: gene therapy trial on track, and promising data in animals

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.

Dr Leora FoxApril 15, 2021

Caution urged for the use of gene-editing technology CRISPR

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

Dr Rachel HardingAugust 12, 2020

Exciting new Huntingtin lowering tool described

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.

Dr Jeff CarrollAugust 05, 2019

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients

Dr Jeff CarrollJanuary 30, 2019

Advances on many fronts in the battle against the protein that causes Huntington's disease

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

Dr Jeff CarrollDecember 04, 2018

New roles for huntingtin: removing a healthy protein to understand its function

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.

Dr Leora FoxSeptember 26, 2017

Important advances in next generation genome editing tools for Huntington's Disease

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

Mr. Shawn MinnigOctober 31, 2016

Could a new "jaw-dropping" breakthrough help treat Huntington's Disease?

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?

Dr Jeff CarrollFebruary 25, 2016