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Human Respiratory System Lungs Anatomy

New Nanoparticles Carrying mRNA Can Perform In-Lung Gene Editing

A newly created nanoparticle that carries a messenger RNA (mRNA) payload that can be administered to the lungs may offer a method to provide an inhalable treatment for cystic fibrosis and other lung diseases.
Cardiovascular disease

Gene Editing Offers Heart Disease Hope

CRISPR gene editing could offer a new way to prevent cardiac damage following a stroke or heart attack, research suggests.
CRISPR - Cas9 system

Human Genetic Diversity Could Alter Off-Target Gene Editing Effects

Computational methods could help make CRISPR gene editing safer for therapeutic use in more diverse populations by helping to prevent off-target effects.
Digital DNA. Biotechnology concept. 3D render

Ionis and Metagenomi Partner Over Gene Editing

Antisense-deveoper Ionis Pharmaceutical and gene editing company Metagenomi have entered a collaboration to pursue investigational drugs for genetic targets.
Schematic illustration of the intended therapeutic effect of intravenously administered PNA-based gene editing nanoparticles: to ameliorate CF in multiple affected organs by correcting the underlying genetic mutation.

Gene Editing Shows Potential for Cystic Fibrosis

A single injection that deploys gene editing technology can correct a common mutation that causes cystic fibrosis and improve lung and gastrointestinal symptoms, research in mice has revealed.
3d illustration of a part of an RNA chain to illustrate new RNA therapeutics

RNA and Gene Editing Pioneers Launch Orbital Therapeutics

RNA medicines startup Orbital Therapeutics launches with backing from Beam CSO and Alnylam founder, among other high-level scientists.
Image of several pink mitochondria on a black background to illustrate how fumarate, a chemical byproduct of mitochondrial metabolism, is known to trigger inflammation in the absence of the enzyme fumarate hydratase

TALED Gene Editing Could Help Target Mitochondrial Diseases

A new type of mitochondrial gene editing shows promise for editing mutations that cause disease and could help correct them in the future.

Editas Announces First-Ever Pediatric In Vivo CRISPR Gene Editing Medicine Administration

Prior to the start of pediatric testing of EDIT-101, Editas had completed dosing of all adult cohorts in its BRILLIANCE study. The clinical results from this arm of the trial demonstrated a favorable safety profile and encouraging signals of clinical benefit.
3D rendered Illustration, visualisation of a anatomically correct Mitochondrion, a organelle of most eukaryotic and other cells - to illustrate mitochondrial RNA changes in cancer

Gene Editing Used to Repair Mitochondria

Researchers have used gene editing to modify the mitochondria in live mice, potentially paving the way for treatment of many rare diseases.
Genetic engineering concept. DNA. Gene therapy. Medical technology

Gene Editing Biotech Metagenomi Achieves $175M Series B

Metagenomi says the oversubscribed $175 million Series B financing it completed Tuesday will enable it to advance its lead in vivo and ex vivo gene editing therapy programs into the clinic.


Inside Precision Medicine