Using Lipid Nanoparticles to Efficiently Deliver CRISPR/Cas9 for Genome Editing

S. C. Alexander et al. Transthyretin amyloidosis (ATTR) is a progressive disease caused by accumulation of amyloid deposits of misfolded transthyretin (TTR) protein in multiple tissues including the heart, nerves and gastrointestinal tract. Reduction of TTR monomer via stabilization of circulating tetramer and silencing of TTR gene expression in hepatocytes of ATTR patients have emerged […]

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

fapnewstoday.com Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. … Minssen said the rapidly growing global market for CRISPR/Cas9 technology could reach $10 […]

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 […]

Humans as Bioreactors

Spartacus In the case of the COVID-19 vaccines, the media and the medical establishment tried getting around this by arguing that since the vaccines did not change the recipient’s DNA, that meant that they weren’t gene therapy. The introduction of foreign nucleic acids into the body to generate foreign proteins is, by definition, gene therapy, regardless […]

Effects of N1-Methyl-Pseudouridine in the Pfizer mRNA Vaccine

Daniel B. Demers The use of messenger RNA (mRNA) vaccines has been developing since 1990.  Historically, there have been three significant problems associated with mRNA vaccines.  First, it has always been a challenge for vaccine developers to get the desired mRNA into the cells of choice (the delivery problem).  Second, introducing a foreign RNA (the vaccine mRNA) […]

Intracellular Reverse Transcription of Pfizer BioNTech COVID-19 mRNA Vaccine BNT162b2 In Vitro in Human Liver Cell Line

white and black labeled bottle

Markus Aldén et al. Preclinical studies of COVID-19 mRNA vaccine BNT162b2, developed by Pfizer and BioNTech, showed reversible hepatic effects in animals that received the BNT162b2 injection. Furthermore, a recent study showed that SARS-CoV-2 RNA can be reverse-transcribed and integrated into the genome of human cells. In this study, we investigated the effect of BNT162b2 […]