Genomics
Gene editing, CRISPR, synthetic biology, personalized medicine, longevity research
Prime Editing-Installed Suppressor tRNAs for Disease-Agnostic Genome Editing
Disease-agnostic prime editing using suppressor tRNAs addresses ~30% of rare genetic diseases caused by nonsense mutations
CRISPR Gene Activation Without Cutting DNA
Epigenetic editing reactivates silenced genes by removing methyl groups without DNA cuts — safer Sickle Cell treatment path
First-in-Human CRISPR Trial Safely Lowers Cholesterol and Triglycerides
Single CRISPR infusion targeting ANGPTL3 reduced LDL ~50% and triglycerides ~55% within 2 weeks, sustained 60+ days
CRISPR-GPT: AI-Powered Gene Editing Copilot
LLM copilot for gene editing trained on 11 years of expert data — enabled first-attempt success by novice researchers
Design of Highly Functional Genome Editors by Modelling CRISPR-Cas Sequences
First fully AI-designed CRISPR gene editor (OpenCRISPR-1) successfully edits human genome — 95% reduction in off-target edits vs SpCas9
FDA Greenlights Life Biosciences' Human Study on Partial Epigenetic Reprogramming
FDA greenlights the first human trial of partial epigenetic reprogramming (Yamanaka OSK factors) — indication is glaucoma + NAION, ~12 patients, starts 2026
Lonvoguran Ziclumeran — In Vivo CRISPR Gene Editing in Hereditary Angioedema (Phase 3 HAELO)
First in vivo CRISPR gene editing therapy to succeed in a Phase 3 trial — a single 50 mg infusion of lonvo-z cut hereditary angioedema attacks 87% vs placebo (p<0.0001); rolling BLA underway, US launch targeted 1H 2027
Bioinformatics Frameworks for Single-Cell Long-Read Sequencing: Unlocking Isoform-Level Resolution
Review establishes single-cell long-read sequencing (SCLR-seq) as mature — PacBio HiFi at 99.9-99.95% accuracy and ONT approaching 99% now resolve full-length isoforms per cell, moving transcriptomics beyond gene-level counts to differential isoform expression
In Vivo Cardiac Prime Editing Corrects RBM20 Mutation in Humanized Mouse Model
First in vivo cardiac prime editing platform with tissue specificity — efficient editing in heart, no detectable editing in liver. Dual-AAV RBM20 prime editing therapeutic rescues cardiomyopathy in humanized mouse model
Multiplexed In Vivo Base Editing Identifies Functional Gene-Variant-Context Interactions
Multiplexed in vivo base editing as functional genomics platform — systematic identification of gene-variant-context interactions in vivo. Bridges in vitro variant screens to whole-organism context
Toward Precision Longevity: Aging Interventions in the Single-Cell Atlas Era
Review argues longevity interventions must move from whole-organism to single-cell precision — cell-type-specific aging patterns are masked by bulk analysis, and systemic drugs (rapamycin) help some tissues while harming others
This Method to Reverse Cellular Aging Is About to Be Tested in Humans
Deep expert-sourced critique of the Life Biosciences Yamanaka trial — surfaces specific cancer, immunogenicity, and systemic-aging concerns that frame how to read the Phase 1 readout
Vertex Presents First-Ever CASGEVY Data in Children Ages 5-11; 1H 2026 Global Submissions
First clinical data for CASGEVY in children ages 5-11 with severe SCD/TDT. Vertex plans 1H 2026 global regulatory submissions for pediatric expansion
Base Editing Breaks Through in the Clinic — Beam Therapeutics 2026 Pipeline (PKU, AATD, Sickle Cell)
Beam's in vivo base editing advances across three indications — FDA clears the BEAM-304 IND for PKU (Jun 2026); BEAM-302 shows first-ever genetic correction in AATD with durable dose-dependent AAT increases; BEAM-101 cuts sickle-disease protein >90% sustained without busulfan