Gene therapies serve as a platform technology that utilizes the same modification method for different targets in the patient genome without the drug delivery limitations of protein-based drugs and the cost of drug development. In contrast to protein-based drugs that require weekly infusion, gene therapy modifications provide a long lasting treatment for disease. Combining this technology with knowledge of the human genome can potentially treat a vast number of rare inherited genetic disorders.
Recombinant, replication-defective viral vectors were the first molecular tool enabling efficient, nontoxic gene transfer into human somatic cells.1 Preclinical and clinical successes using viral vectors, such as Glybera and Luxturna, for mediated gene replacement, gene silencing and gene editing have demonstrated precedent success.2
RNA virus
Carrying capacity of about 9 kilobases (kb)
Integrates into host genome 3
Linear, single-stranded DNA
Carrying capacity of about 4kb of inserts
Recombinant strain does not integrate into host genome 3
Innate ability to infect mammals
Does not integrate into host genome
Large DNA payload due to its large genome size (150 kb)
Established targets in the viral genome that can be deleted without affecting viral functions 3