Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

Infants Diagnosed with Spinal Muscular Atrophy and 4 SMN2 Copies through Newborn Screening – Opportunity or Burden?1

“…AAV based SMA gene therapy (Onasemnogene abeparvovec) is already approved in US for children below 2 years [54] and it is expected to be approved by the EMA in 2020. Furthermore, the older the children are at the time of application the higher is the risk that they have developed antibodies against scAAV9 and therefore effectively precludes this special therapy [55].…”

Materials promoting viral gene delivery

“…33 Adeno-associated viruses (AAV) have emerged as promising vectors with low immunogenicity and long-term stability, resulting in the recently approved therapies, marketed as Luxturna® and Zolgensma®. 34,35 As part of the retrovirus (RV) family, lentiviruses (LV) have been increasingly applied in recent years due to their inherent ability to infect both dividing and non-dividing cells, efficient integration into host genetic information and possibility for large scale production. [36][37][38][39] Finally, oncolytic viruses are attracting growing interest as versatile cancer therapeutics.…”

<p>Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence</p>

“…This must then lead to further translation of the gene into functional proteins without causing insertional mutagenesis, oncogenesis, or an exorbitant host immune response. 23 25 Many factors play a role in determining the efficiency of the DNA expression, including the type of promoter used, the addition of enhancer sequences, and vector capsid components. 26,27 Capsid proteins can be modified to improve transduction, cell targeting, and counter pre-existing immunity.…”