Nature Outlook |

Gene therapy

After a roller-coaster ride of hype and disappointment, the decades-long effort to cure diseases by repairing or replacing faulty genes is starting to yield useful treatments. Diseases that have defied treatment could be reversed by a one-time fix to a faulty gene.

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Features and comment

Gene tinkering opens the door to treatments for an array of diseases

Outlook | | Nature

Gene therapy could one day be used for bodily enhancement, creating an ethical minefield for physicians, says Ellen Wright Clayton.

Outlook | | Nature

Anti-seizure medication doesn’t work in every person with epilepsy. But a treatment option is emerging that would spare the need for brain surgery.

Outlook | | Nature

Insertion of genetic information can prompt the body to make antibody-based drugs, offering a fresh approach to treating diseases such as influenza, as well as infections like HIV.

Outlook | | Nature

The hope of gene therapy could be crushed by its financial burden unless there are more rational ways of paying for it, says Michael Sherman.

Outlook | | Nature

More from Nature Research

A glutamate-gated chloride channel delivered via gene therapy is shown to detect elevated brain glutamate levels and trigger the suppression of neuronal excitability, thereby attenuating seizure activity in two rodent models of epilepsy.

Letter | | Nature Medicine

In utero GBA gene therapy extends lifespan and provides long-lasting phenotypic amelioration in a mouse model of neuronopathic Gaucher disease. Fetal ultrasound-guided in utero gene vector delivery is also achieved in the non-human primate brain.

Letter | | Nature Medicine

The potential of adeno-associated viral (AAV)-mediated gene therapy for neurological disorders is rapidly emerging. Evidence of clinical efficacy and safety, as well as durable transgene expression, has now been reported in several central nervous system disorders. Here, Sah and colleagues discuss key considerations in the design and development of therapeutic AAV vectors, highlighting promising therapeutic targets and recent clinical trials.

Review Article | | Nature Reviews Drug Discovery