Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions

Research output: Contribution to journalReview articlepeer-review

64 Scopus citations


Adeno-associated virus (AAV) vector-based gene therapy is currently the only in vivo gene therapy approved in the US and Europe. The recent tragic death of three children in a clinical trial to treat X-Linked Myotubular Myopathy by delivering myotubularin with an AAV8 vector notwithstanding, AAV remains a highly promising therapeutic gene delivery platform. But the successful use of AAV vectors to treat an increasing number of diseases also makes establishing protocols to determine therapeutically relevant titers of pre-existing anti-AAV antibodies and approaches to deplete those antibodies more urgent than ever. In this mini review, I will briefly discuss (i) our knowledge regarding the prevalence of anti-AAV antibodies, (ii) the challenges to measure those antibodies by methods that are most predictive of their influence on therapeutic efficacy of AAV gene transfer, and (iii) approaches to overcome the formidable hurdle that anti-AAV antibodies pose to the successful clinical use of AAV gene therapy.

Original languageEnglish
Article number658399
JournalFrontiers in Immunology
StatePublished - 17 Mar 2021


  • aav
  • adeno-associated virus
  • antibodies
  • antibody assays
  • gene therapy
  • prevalence of antibodies
  • removal of antibodies
  • removal of inhibitors


Dive into the research topics of 'Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions'. Together they form a unique fingerprint.

Cite this