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PETBioNewsGlossaryGene therapy

BioNews

Gene therapy

The use of genetic material (DNA or RNA) as a medical treatment to either: replace (or repair) a faulty or missing gene product; switch off production of a harmful gene product; or destroy specific cells involved in disease.

378 articles
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
1 April 2022 • 2 minutes read

New gene therapy for patients with blistering skin disorder

by Joseph Hawkins

Patients with a rare genetic skin condition have been successfully treated, for the first time, using a novel gene therapy...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
18 February 2022 • 2 minutes read

Tay-Sachs treated with gene therapy for first time

by Dr Ara Schorscher-Petcu

Gene therapy given to children affected by the fatal neurological disorder Tay-Sachs disease is safe and stabilises disease progression, reports an exploratory proof-of-concept study...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
Reviews
4 February 2022 • 5 minutes read

Event Review: Dreams and Nightmares of the Genetic Age

by Anna Wernick

In his loud shirt, covered in green leaves and plants, the University of Manchester's professor of Zoology, Matthew Cobb, was dressed appropriately for this evening's talk...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
4 February 2022 • 2 minutes read

Leukaemia patients still in remission a decade after CAR-T gene therapy

by Farah Alam

Two patients who received CAR-T gene therapy over a decade ago are still in remission, the longest follow-up study of its kind has shown...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
21 January 2022 • 2 minutes read

Newly discovered protein essential for CRISPR/Cas3 genome editing

by Tom Turner

A hidden component of the CRISPR/Cas3 genome editing system, which is essential for it to work, has been discovered by scientists...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
7 January 2022 • 2 minutes read

Gene therapy developed to treat limb-girdle muscular dystrophy

by Dr Holly Barker

A new gene therapy approach has shown promise in a preclinical mouse model for treating limb-girdle muscular dystrophy 2B, a rare genetic disorder which causes severe muscle weakness...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
Comment
12 November 2021 • 4 minutes read

A 'serious' threshold for genomic technologies – context counts!

by Marie-Christine Roy and 3 others

The determination of how 'serious' a condition is has direct and important implications for policy and clinical translation of existing and emerging genomic technologies...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
5 November 2021 • 2 minutes read

RNA-based control switch could be used to turn on gene therapies in cells

by Anna Wernick

A new technique to turn therapeutic genes on in specific cells has been developed, potentially changing the future of gene therapies...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
5 November 2021 • 2 minutes read

Gene therapy enhances the effects of Parkinson's disease drug in mice

by Paige Mumford

Gene therapy delivered to a novel mouse model of Parkinson's disease improved the effects of existing Parkinson's drug levodopa...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
29 October 2021 • 2 minutes read

American organisations join together to develop gene therapies

by David Cansfield

The National Institutes for Health, Food and Drug Administration and 15 pharmaceuticals and non-profit organisations have joined together to accelerate the development of gene therapies in the USA...

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