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Several years in the making but finally in print! Thank you to those who have contributed chapters to this book!

nytimes.com/2022/06/05/hea… “I believe this is the first time this has happened in the history of cancer.” Rectal tumors disappeared in every patient

Intellia shares positive interim data from its ongoing Phase 1 study of NTLA-2001 for the potential treatment of #transthyretinamyloidosis (#ATTR) at @EASLNews. See link to full press release: bit.ly/3QLwhcz #CRISPR

#RNATx2022 Paloma Giangrande Wave Life Sciences: GalNAc-AIMers: A-to-I editing oligonucleotides Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides pubmed.ncbi.nlm.nih.gov/35256816/

#RNATx2022 Bob Brown @UMassMedical: Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis pubmed.ncbi.nlm.nih.gov/35075293/

Maybe the first #CRISPR paper you read? Don't know it? Time travel to read: @MartinJinek et al. Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity. Online 10 years ago science.org/doi/10.1126/sc… Covered in @NYTimes nytimes.com/2022/06/27/sci… #CRISPR News

Surprise! After local delivery of ASOs to the lung, the most robust gene silencing is seen in fibroblasts. This work led by Minwook Shin is online today at NAR. academic.oup.com/nar/advance-ar… @otsociety Collab Manuel Garber Rene Maehr KhvorovaLab ErikSontheimerLab RNA Therapeutics Institute

Out today is our latest work on the mechanism of DCBs #drugcoatedballoon Published at journals.sagepub.com/doi/full/10.11…

We’re pleased to report positive results from the Phase 2 study of cemdisiran, our investigational #RNAi therapeutic in development for the treatment of #IgANephropathy in collaboration with Regeneron. #RNAiTherapeutics #siRNA

Drinking 2 - 10 or more cups of tea a day is associated with a lower risk of death from all causes but a proportionally higher percent of one's life spent in the bathroom acpjournals.org/doi/10.7326/M2…

Now online: a review by the brilliant Samantha Sarli, PhD introducing the diverse range of nucleic acid therapeutic technologies - featured in a special issue on space applications, but worth a read for anyone new or looking to understand the field. Oligonucleotide Therapeutics Society sciencedirect.com/science/articl…

On our blog: Paloma Giangrande explains ASGCT’s comments on the proposed update to the Screening Framework Guidance for Providers of Synthetic Double-Stranded DNA. bit.ly/3B8orD2

Looking forward to deliver !!

Day 3 will start with the preclinical session chaired by Aurélie Goyenvalle and Martin Maier. First talk is given by Chandra Vargeese from Wave life sciences. She will talk about backbone chemistry.

Ronald Buijsen She discussed multiple ways stereopure chemistry can be exploited: dna editing, gene upregulation, transcript reduction and (my favorite) exon skipping. Wave Life Sciences

🚨New paper in Nature Chemistry w/ Phil Holliger joint 1st w/ brill saxophonist & bowtie fan Niklas Freund We describe a polymerase for efficient DNA-templated synthesis of 2’OMe- & MOE-RNA & develop biostable aptamers & RNA-cleaving 2’OMezymes #synbio nature.com/articles/s4155…

News from around the RNA world: new avenues in RNA biology, biotechnology and therapeutics from the 2022 SIBBM meeting journals.biologists.com/bio/article-ab…

Intrathecal ASO can cause hydrocephalus in patients, in addition to their intended therapeutic effects. New therapeutics often have new toxicities. Understanding these should help in advancing the great promise of ASO technology for other patients. nytimes.com/2022/10/26/hea…

A hormone produced by muscles during vigorous exercise may one day be used as a drug to treat excessive alcohol use. The FGF21 hormone, when given as a drug, reduces alcohol consumption by 50% in monkeys. This underscores how important EXERCISE is for curbing drinking behavior.

Day 1 #OTS23…already to a great start! Fantastic Educational Session on Innovating and Commercializing next gen oligo therapies!