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Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease.
The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver — are infused into the bloodstream. In the trial, six people with a rare and fatal condition called transthyretin amyloidosis received a single treatment with the gene-editing therapy. All experienced a drop in the level of a misshapen protein associated with the disease. Those who received the higher of two doses tested saw levels of the protein, called TTR, decline by an average of 87%.
videos & visualsAlnylam Clinical Trial Update – ASG Webinar 4/11https://www.youtube.com/watch?v=K2TmwAfu...
videos & visualsAmyloidosis — Why Did I Have to Get All of Those Tests – ASG Webinar 4/7https://www.youtube.com/watch?v=IzC_yaqs...
videos & visualsWild-Type Transthyretin Cardiac Amyloidosis – Mayo Clinichttps://www.youtube.com/watch?v=RnWxzRD1...
educationHereditary ATTR Val122Ile Cardiac AmyloidosisHereditary ATTR amyloidosis is an inheri...
educationPatisiran, an RNAi Therapeutic, for Hereditary Transthyretin AmyloidosisBackground: Patisiran, an investigation...
news & meetingsGene Editing Offers Hope for Progressive Cardiomyopathy Due to AmyloidosisInvestigators have found that intravenou...
news & meetingsCRISPR Gene Editing Reduces Disease-Causing Protein in Hereditary Transthyretin AmyloidosisThe results of the phase 1 study open-la...
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