Retinal gene therapy trial shows positive result
A clinical trial using gene therapy for treating a common cause of genetic blindness published positive results in Nature Medicine...
BioNews
Vector
A delivery system used in the laboratory to carry foreign DNA into a cell. Modified viruses are commonly used as vectors in gene therapy.
96 articles
New FDA guidelines for gene therapy products
The US Food and Drug Administration has released seven new guidelines that will help scientists safely develop novel gene therapy products...
NHS to provide gene therapy for rare eye disease
An in-body gene therapy treatment has been approved to treat a rare genetic form of blindness...
Scientists insert human genes into monkey brains
Monkeys with an inserted human gene involved in brain development performed better on cognitive tasks, a new study has found...
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.
First in-body genome editing trial delivers mixed results
The first in vivo genome editing therapy has been successful in editing patients' DNA, but has not shown a clinical benefit...
CRISPR used to block HIV virus replicating in cells
Scientists in Japan have harnessed genome editing to successfully block the HIV virus from replicating inside infected cells....
Promising results from beta-thalassaemia gene therapy trial
A clinical trial that used a new gene therapy to treat beta-thalassemia has shown very promising results...
New RNA editing tool shows promise for dementia
Researchers in the USA have developed a new RNA editing approach, which could be beneficial in treating dementia...
High-dose gene therapy appears toxic in monkeys and piglets
Animals given high doses of gene-carrying viruses in a new gene-therapy trial showed signs of severe toxicity.
Human immune response may stymie CRISPR genome editing therapies
The human adaptive immune response against the Cas9 protein - part of the CRISPR genome editing system — may pose a barrier to new therapies, suggests a new study...