Google
Trusted Resources: News & Meetings
Latest announcements and gatherings
CRISPR Gene Editing Reduces Disease-Causing Protein in Hereditary Transthyretin Amyloidosis
The results of the phase 1 study open-label trial showed the therapy was safe, raising the possibility of a more effective and more appealing treatment for the progressive and fatal disease, in which amyloid builds up in the body’s organs and tissues, where it can cause loss of sensation, heart problems, or other symptoms. Life expectancy after diagnosis is about seven to 12 years. Current treatment involves long-term therapy to stabilize the protein and slow the formation of amyloid or inhibit TTR protein production by degrading its mRNA, thereby slowing the formation of amyloid.
The new therapy, called NTLA-2001, was given once to six patients at 0.1 mg/kg to half and 0.3 mg/kg to the other half. After 28 days, the concentration of TTR protein in the serum was reduced by 52 percent in those receiving the lower dose and by 87 percent in those receiving the higher dose.
myBinderRelated Content
-
Evolving Landscape in the Management of Transthyretin AmyloidosisTransthyretin (TTR) amyloidosis (ATTR am...
-
He Inherited A Devastating Disease. A CRISPR Gene-Editing Breakthrough Stopped ItPatrick Doherty had always been very act...
-
Alnylam Clinical Trial Update – ASG Webinar 4/11https://www.youtube.com/watch?v=K2TmwAfu...
-
Clinical Presentation, Diagnosis and Treatment of TTR AmyloidosisSystemic amyloidosis can be hereditary o...
-
FDA Approves Patisiran Infusion for Hereditary Transthyretin-Mediated AmyloidosisThe FDA announced that it has approved p...
-
Taking Your Next Steps: Life Following hATTR Amyloidosis DiagnosisBeing diagnosed with hATTR amyloidosis...
-
Inotersen Treatment for Patients With Hereditary Transthyretin AmyloidosisBackground: Hereditary transthyretin am...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences.
More information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Community Key Collaborator
Support for this landing page is provided by an independent education grant from
Translation of this platform made possible through an independent education grant from
Powered by
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences.
More information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
