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Gene Therapy & Genomic Medicine
CRISPR edits, delivery vectors, rare‑disease trials, and approvals.
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Latest in Gene Therapy & Genomic Medicine
Beth Jacob analyzes ethics of genetic engineering in three programs Oct. 21-25
1+ hour, 52+ min ago (583+ words) A poster for the 1997 science fiction thriller, Gattaca. When does science fiction become science fact, and what does that mean for the day to day lives of ordinary folks? Those are questions people will grapple with in Scientists in Synagogues,…...
Exonuclease Boost Makes CRISPR Cas12a Cut Big Chunks Out of Sugarcane’s Daunting Genome
57+ min ago (711+ words) Bioengineer.org...
Researchers find STRIKE route for siRNA kidney disease treatment
4+ hour, 34+ min ago (918+ words) Judo Bio showcased a ligand-siRNA conjugate that could turn the tables in treating kidney disease. The kidneys remain an elusive target for RNA-based therapies. While siRNAs and other oligo drugs often end up in clearance organs due to blood filtration,…...
Scientists Create First Hypoallergenic Gene-Edited Dogs
6+ hour, 26+ min ago (194+ words) The European Times They âswitched offâ the gene responsible for producing the primary allergen found in dog saliva, fur, and dander To create the two dogs, scientists edited the gene responsible for producing the main allergenic protein in dogs…...
PacBio and TychoBio partner to advance AI-driven RNA therapies
11+ hour, 40+ min ago (345+ words) PacBio and AI drug discovery company TychoBio have announced a collaboration to generate large-scale full-length RNA datasets to support the development of new RNA therapies for rare diseases. The companies plan to use PacBio HiFi sequencing and Kinnex long-read RNA…...
A new, robust, long-lasting, and safe nucleic acid therapeutic
10+ hour, 21+ min ago (203+ words) Researchers have developed a novel nucleic acid therapeutic platform that achieves both high efficacy and safety against diseases of the central nervous system Credit: Institute of Science Tokyo Mechanistic studies revealed that Chol-HDO[PMO] achieves improved potency by binding to…...
Researchers at OHSU Working on New Way to Cure Rare Kidney Conditions
10+ hour, 9+ min ago (128+ words) PORTLAND, OR – Researchers at Oregon Health and Science University are getting federal funding to advance gene therapy for rare kidney conditions. These conditions currently have no known cures, according to Dr. Hiroyuki Nakai at OHSU. “It delivers healthy a healthy…...
An RNA mark for pluripotency
18+ hour, 40+ min ago (158+ words) Nature Chemical Biology (2026) Cite this article A chemical modification of 7SK RNA links RNA modification to chromatin remodeling during human cell reprogramming. PUS7 installs this modification, enabling reprogramming without exogenous OCT4 and providing an alternative route to induced pluripotency. Takahashi, K. & Yamanaka, S. Cell 126, 663–676 (2006). Polo,…...
Nanowire Retinal Implant Restores Vision and Sees Infrared
13+ hour, 2+ min ago (820+ words) Summary: Researchers have developed a retinal prosthesis made of tellurium nanowires that restores partial vision in blind mice and enables near-infrared vision in primates. The implant, woven into a lattice of light-sensitive nanowires, converts both visible and near-infrared light into…...
Can we grow accurate biological versions of human embryos?
15+ hour, 39+ min ago (594+ words) Original story from the University of Sydney (Australia). The answer is not yet, but we’re getting closer. Researchers have developed a computational framework to test how closely lab-grown models resemble real human embryos. University of Sydney (Camperdown, Australia) researchers have…...