Gene tinkering opens the door to treatments for an array of diseases
Nature Outlook |
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
Cancelling out an illness before a child is born offers the most potential benefit.
Gene therapy could one day be used for bodily enhancement, creating an ethical minefield for physicians, says Ellen Wright Clayton.
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.
The research is promising, but a true cure for this painful condition could be years away.
Gene therapy for single-gene disorders is at a pivotal period in its evolution, with continued successful development requiring tight collaboration among industry, academic, regulatory, clinical and patient communities.
The largest organ in the body is a prime target for gene therapy.
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.
Researcher and entrepreneur Luk Vandenberghe thinks he can transport genes into cells much more efficiently by improving the viral vectors that carry them.
The medical regulatory authorities ride a wave of clinical studies for gene therapies.
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.
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Two recent studies describe clinical successes for single-dose gene therapy in trials for two forms of hemophilia.
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.
Patients with junctional epidermolysis bullosa (JEB) carry mutations in genes that encode components of the basement membrane, which ensures the integrity between the epidermis and the dermis, such as laminin-332. These mutations cause blistering of the skin and chronic wounds. Following initial treatment of an adult patient with a limited affected region, Michele De Luca and colleagues reconstruct the full epidermis of a 7-year-old patient with autologous transgenic cells transduced with a virus vector carrying the non-mutated form of laminin-322. The integration sites of the virus used for gene delivery provide a tracing tool ex vivo and in vivo and demonstrate that the human epidermis is sustained by a limited number of long-lived stem cells.
Efficient gene transfer to the mouse inner ear is achieved with a synthetic adeno-associated viral vector.
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.
The long-term follow-up results of a phase 1/2 retinal gene therapy clinical trial for choroideremia (
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.