Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA
fapnewstoday.com The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NTLA- 2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin (ATTR) amyloidosis. … The aim of NTLA-2001 is to disrupt the TTR gene, thus decreasing transthyretin production and preventing the buildup of these harmful protein […]
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
Julian D. Gillmore et al. Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart. NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It […]
GMO: Myth & Facts
What They Don’t Want to Tell You About Genetically Modified Crops and Foods Claire Robinson The public is constantly told that genetically modified (GM) crops and foods are needed to feed the world’s growing population and to meet the challenges that face farmers – climate change as well as pests and diseases. It is claimed […]
Yikes: The Brave New World of Genetically Modified People
Tessa Lena This story is about the hubris of conquest and genetic modification. Let’s start with the ugly. The richest people of the world, as well as their loyal servants from the World Economic Forum, are on the Genetic Modi cation team. According to the World Economic Forum, “the evolution of gene testing and gene […]
