CRISPR delivered through the nose edits brain genes in mice with ALS mutations

Key Findings

🫁 Inhaled gene editing fixes cystic fibrosis mutations in mouse lungs

• Researchers created 960 different amino acid-derived lipid nanoparticles and identified CHCha-10 as the most effective for lung delivery

• The nanoparticles delivered base editor mRNA that corrected the CFTR G542X mutation, restoring chloride channel function in cystic fibrosis mice

• Intratracheal administration showed enhanced mucus penetration and epithelial-specific transfection in both mice and ferrets

🔬 Miniature gene editor fits in single virus for cancer immunotherapy

• Scientists engineered TnpB, a compact alternative to CRISPR-Cas9, with a minimized 93-nucleotide guide RNA that increased gene expression by 2,889-fold

• A single adeno-associated virus delivered the system to activate immune genes CXCL9, IL-15, and IFN-γ in cancer cells

• The treatment enhanced T cell migration and killing of cancer cell lines, achieving 60% tumor elimination (3 out of 5 tumors) when combined with anti-PD-1 therapy in humanized mice

💊 Water-based nanoparticles deliver gene editing without toxic solvents

• Researchers developed lipid nanoparticles using only water, eliminating the need for ethanol and polyethylene glycol (PEG) commonly used in current formulations

• The solvent-free nanoparticles efficiently transfected primary human immune cells and delivered multiple components for CRISPR-Cas9 applications

• In vivo testing showed preferential targeting of tissues outside the liver, unlike conventional nanoparticles that primarily accumulate in liver tissue

🎯 Smart CRISPR system switches between editing modes with drug triggers

• Scientists created a controllable base editing system that switches between four modes (OFF, cytosine editing, adenine editing, or both) using small molecule drugs

• The system achieved up to 43.4% C-to-T editing or 42.9% A-to-G editing at four human gene sites when activated

• In a screen of 4,000 guide RNAs, researchers identified four amino acids in monkey CD163 that reduced highly pathogenic virus replication by more than 100-fold

🧬 FDA framework speeds approval for ultra-rare disease gene therapies

• The FDA published a new "plausible mechanism framework" in February 2026 for faster approval of individualized genetic therapies

• Researchers developed a customizable prime editing platform targeting 7 urea cycle disorders and met with FDA to discuss an "umbrella-of-umbrellas" clinical trial

• The approach could expedite approvals for ultra-rare diseases by allowing treatments for multiple related conditions under one regulatory pathway

🌱 Plant gene editing creates herbicide resistance and improves iron content

• CRISPR-Cas12a variants with added DNA sequences (introns) showed substantial improvement in cytosine base editing efficiency in rice compared to standard versions

• Base editing successfully introduced herbicide-resistant mutations in rice OsACCase gene and enhanced iron loading in wheat grain scutellum

• Whole genome sequencing confirmed the improved editors rarely introduced unwanted mutations at off-target sites