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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.
This Nature Outlook is editorially independent. It is produced with third party financial support. About this content.
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.
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.
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.
Autologous transgenic epidermal stem cell cultures are used to reconstitute almost the entire epidermis of a patient with severe junctional epidermolysis bullosa.
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 (NCT01461213) support the safety and efficacy of the treatment.
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.