Blog articles

In vivo and ex vivo gene therapies explained

Gene therapies can take place either 'in vivo' or 'ex vivo'; we compare these two approaches and their importance in gene therapy
A computer manipulated photo of a hand holding a pair of self-locking medical tweezer scissors and DNA helixes. The tweezer scissors are removing a small red section that is on one of a DNA helixes.

Hereditary angioedema gene therapy hope

A CRISPR-based genome therapy has shown promising results for patients with a rare genetic swelling disorder
Conceptual illustration of neuron cells with glowing link knots in abstract dark space, high resolution 3D illustration

Clinical trials show promise for first ‘gene silencing’ treatment for Alzheimer’s disease

A new RNA-based therapy for Alzheimer’s disease that's in clinical trial phase could be the first of its kind to use ‘gene silencing’ for the condition

World-first gene therapy trial for Hunter syndrome opens

We explore a new treatment that aims to cross the blood-brain barrier for the first time in Hunter syndrome
A strand of stylised blue DNA on a plain background with a magnifying glass focusing on a section of the DNA strand

Casgevy – how it works

In this week’s article, we revisit Casgevy and explore its utility and application in healthcare
An illustration of a multicoloured DNA strand running vertically down the centre of the image with two hands on either side of it. The hands are wearing blue surgical gloves. One hand is holding tweezers that are gripping the DNA strand and the other hand is holding a scalpel which has sliced the DNA strand.

Casgevy – the first CRISPR therapy

The UK has approved a new type of gene therapy for the blood disorders sickle cell disease and beta-thalassaemia, a world-first using CRISPR technology
A graphic of an ear in isolation on a blue background. There are white and grey DNA strands going diagonally across the background

Gene therapy – a new approach for paediatric hearing loss

A new gene therapy is being trialled for children with a rare form of deafness caused by variants in the OTOF gene
A baby in a blue sleep suit lies inside an incubator.

A ground-breaking new gene therapy has saved the life of its very first NHS patient

A baby girl is showing no further signs of the fatal genetic disorder metachromatic leukodystrophy after treatment with an exciting new gene therapy
An illustration of a multicoloured DNA strand running vertically down the centre of the image with two hands on either side of it. The hands are wearing blue surgical gloves. One hand is holding tweezers that are gripping the DNA strand and the other hand is holding a scalpel which has sliced the DNA strand.

What are genome editing and gene therapy?

Gene therapy and genome editing technologies are increasingly being reported on in the news and discussed in medical literature, but what are they?

Achromatopsia gene therapy partially restores children’s colour vision

A new gene therapy for achromatopsia has partially restored colour vision in two children, according to new research
Adult holding baby's hand in hospital

NICE approves world-first MLD therapy

We explore a new gene therapy for a rare, fatal condition that affects young children

News articles

New horizon-scanning webinar series for 2023

The GEP has launched the first of an exciting series of expert webinars designed to create a link between academia and genomics education