News on Gene Replacement Therapy

Gene Therapy Comes of Age

Dunbar et al. Science. 2018;359(6372)

The year 2017 marked an exciting year of gene therapy approvals in the US and EU; several promising candidates are in development for the coming years. Despite challenges, concurrent advances in virology, immunology, cell biology, and genetics have propelled gene therapy from an idea to a reality in just under 50 short years.1

Manufacturing of Recombinant Adeno-Associated Viral Vectors for Clinical TrialS

Clément et al. Mol Ther. 2016;3:16002

Recombinant adeno-associated virus (rAAV)-based vectors are widely used for the delivery of gene therapy due to their ability to elicit robust and long-term transgene expression with minimal toxicity and immunogenicity. There is a need to establish methods for high-yield production and purification systems for clinical grade rAAVs under good manufacturing practice (cGMP) settings.2

Gene Therapy for the CNS Using AAVs: The Impact of Systemic Delivery by AAV9

Saraiva et al. J Control Release. 2016;241:94-109

Gene therapies that target the cells of the CNS face the difficult challenge of crossing the blood-brain barrier (BBB). Certain AAV serotypes, such as AAV9, have an affinity and an ability to cross the BBB and deliver genetic material into the nucleus of these hard to reach cells.3

Get More Updates About GRT