Arun Srivastava – VJRegenMed

The need for next-gen AAV vectors

admin_vjr_user — Fri, 21 May 2021 17:21:51 +0000

Arun Srivastava, PhD, University of Florida, Gainesville, FL, discusses the need for the development of next-generation adeno-associated virus (AAV) vectors. Since the host immune system is unable to distinguish between naturally occurring AAVs and the first generation of AAV vectors designed for therapeutic gene delivery, it targets both equally. In order to improve the safety and reduce the immune system’s response to AAV-based gene therapies, it is therefore important to develop novel AAV vectors that are distinct from naturally occurring AAVs by modifying the vector capsids. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

NextGen & GenX AAV vectors for DMD gene therapy

admin_vjr_user — Fri, 21 May 2021 17:21:47 +0000

Arun Srivastava, PhD, University of Florida, Gainesville, FL, explains that due to the high volume of muscle is the body, clinical trials investigating first-generation adeno-associated virus (AAV)-based gene therapies for muscular dystrophy require the use of high vector doses, resulting in the need for immune-suppression. In an effort to address this, next-generation (NextGen) AAVrh74 vectors and single-stranded generation X (GenX) AAV vectors have been developed. The encapsulation of the GenX genome containing the micro-dystrophin gene into NextGen AAVrh74 vector capsid, referred to as an optimized (Opt) AAVrh74 vector, is also being investigated and offers the potential to reduce vector doses while maintaining therapeutic efficacy for muscular dystrophy. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Synthetic AAV vectors for gene therapy of hemophilia in children

admin_vjr_user — Fri, 21 May 2021 17:21:44 +0000

Arun Srivastava, PhD, University of Florida, Gainesville, FL, explains that a key challenge associated with the use of adeno-associated virus (AAV) vectors for gene therapy for hemophilia in children is the fact that up until age 10-12, the liver is still growing and dividing, meaning traditional AAV gene therapies are diluted out with every cell division, given their episomal nature. Synthetic AAV vectors containing a no-end (NE) AAV DNA encapsulated in liver-specific synthetic liposomes are currently being investigated. It is hoped that this strategy may allow repeated dosing and specific targeting of the liver, without the induction of a host immune response. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.