Penn Medicine’s Carl June, MD, has won the 2024 Breakthrough Prize in Life Sciences for his work in CAR T cell therapy. T cell therapy marked a transformative moment in medicine & opened new possibilities for personalized therapies for patients worldwide. Congrats, Dr. June!

Polygenic burden of short tandem repeat expansions promote risk for Alzheimer's disease | medRxiv medrxiv.org/content/10.110…

Synthesis of peptide–siRNA conjugates via internal sulfonylphosphoramidate modifications and evaluation of their in vitro activity | Nucleic Acids Research | Oxford Academic academic.oup.com/nar/advance-ar…

Hot off the press! Check out paper from RTI's Khvorova Lab KhvorovaLab cell.com/molecular-ther…

Guangping Gao, PhD, who has dedicated much of his career to developing a gene therapy for Canavan disease, says patients like Noa push him. Read about how UMass Chan Medical School researchers partner with patients to advance research together: direc.to/keir #AdvancingTogether

Amelioration of Tau and ApoE4-linked glial lipid accumulation and neurodegeneration with an LXR agonist: Neuron cell.com/neuron/fulltex…

Circuit-specific gene therapy reverses core symptoms in a primate Parkinson’s disease model: Cell cell.com/cell/fulltext/…

RNA/DNA hybrid duplexes are involved in several important biological processes. 🧬 In a study, published in J. Am. Chem. Soc. , scientists described a novel tool to predict the stability of these structures in living cells. Check out our latest press release: bit.ly/3GmUt1b

Uncovering the functional diversity of rare CRISPR-Cas systems with deep terascale clustering | Science science.org/doi/10.1126/sc…

Jeff Carroll inherited Huntington’s Disease. He researches the disease as an Assoc. Prof. of Neurology at the University of Washington and serves as an n-Lorem Scientific Advisor and member of the ATTC that helps assess submitted patient applications. nlorem.org/inheriting-hun…

Ionis has posted its clinical trial of a PrP-lowering ASO: clinicaltrials.gov/study/NCT06153… Status still "not yet recruiting", study sites not listed. BUT: our first glimpse of details: symptomatic, N=56, double crossover, 24 wks, primary endpoint AEs, secondary endpoints PK & CSF PrP

Become an OTS Member! Select the link below to learn about membership benefits and to apply. oligotherapeutics.org/become-a-membe… #ots #oligotherapeutics #oligonucleotide #biotech #biotechnology #science #research #pharmaceuticals #networking #membership #member #otsmember

To succeed you need to focus on future, be good with people and bring results. hbr.org/2023/07/3-ways…

Time-resolved crystallography captures light-driven DNA repair | Science science.org/doi/10.1126/sc…

Congratulations, Dr. Nathan Bamidele, from ErikSontheimerLab, on his successful thesis defense today! Fantastic talk on Protein and Guide RNA Engineering of Compact Cas9 for Enhanced Precision Genome Editing! Congratulations!

The First FDA Approval for a GalNAc-conjugated ASO ⬇️ oligotherapeutics.org/the-first-fda-… #ots #oligotherapeutics #oligonucleotide #biotech #science #research #currentscience #attramyloidiosis #fda #fdaapproved #GalNAc #amlyoidosis #wainua #amyloidosistreatment #hATTR #pharmaceuticals

Delivery, delivery, delivery! Join us in Siracusa, Italy from April 4-7 to discuss new advances in nucleic acid delivery and how to solve the remaining challenges! Don't miss this opportunity. Abstract deadline is March 15, 2024. Register here: engconf.us/conferences/bi…

.Mariana Lenharo Thank you for covering newer technologies in genetic medicine space. "Move over, CRISPR"?! Please refrain from zero-sum-game-ism. This is the realm of some in the investment community (vide infra) but is below the dignity of @nature. nature.com/articles/d4158…

We are honoured to have Dr. Muthiah (Mano) Manoharan from ⁦⁦Alnylam Pharmaceuticals⁩ give our honorary Bernard Belleau Lectureship ⁦McGill Chemistry⁩ "Living in the World of Nucleic Acids Therapeutics: Challenges and Opportunities”

Terence R. Flotte, MD, told members of Congress today that there is still reason to hope for patients with rare diseases: direc.to/kZed “I am convinced we are going to beat most of these diseases in the end.” #RareDiseaseDay ASGCT Energy and Commerce Committee Energy & Commerce Democrats