Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

The team notes that different forms of SCN1B gene expression may result in different outcomes for the therapy. However, the proof-of-concept is the first step toward a gene replacement therapy for ...