Fierce Biotech

How a new tool for large-scale gene editing could power novel genetic medicines

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

DNA barcoding reveals which gene-therapy nanoparticles reach targets in vivo

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

Forget CRISPR Therapeutics: This Gene‑Editing Player Already Boasts the Profits It Dreams Of

Gene editing, a set of techniques used to alter sections of an organism's DNA, is helping scientists cure diseases previously ...
Earth.com

Canadian man becomes first human cured with prime gene editing

A 19-year-old Canadian man becomes the first human cured through prime gene editing after doctors corrected a rare genetic ...
Morning Overview on MSN

Gene editing advances raise real prospects for future DNA upgrades

The U.S. Food and Drug Administration approved the first cell-based gene therapies for sickle cell disease, including the ...
Fierce Biotech

FDA illuminates new approval pathway for bespoke gene editing therapies

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

Simple 'cocktail' of amino acids dramatically boosts power of mRNA therapies and CRISPR gene editing

Lipid nanoparticles, or LNPs, best known as the delivery vehicle for the COVID-19 mRNA vaccines received by billions of people, are now at the center of a much larger medical revolution. Researchers ...
Technology Networks

Improved Gene Editing Tool Reduces Off-Target Mutations by 80%

A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing ...
Forbes

Gene Editing Or Gene Silencing: The Therapies Poised To Transform Medicine

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

After a baby became the world's first CRISPR gene editing therapy patient, CHOP wants to expand treatment to others

One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand treatment to others.

Studies test whether gene-editing can fix high cholesterol. For now, take your medicine

Today there are injected medicines that block proteins produced by the PCSK9 and ANGPTL3 genes in the liver, thus helping the body clear away cholesterol. The new research uses CRISPR, the Nobel Prize ...
Pharmaceutical Technology on MSN

Gene editing takes centre stage in FDA’s new rare disease approval pathway

The aim of the pathway is to remove red tape for bespoke therapies designed for patients with rare diseases.