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Challenges Posed by Immune Responses to AAV Vectors: Addressing Root Causes.
Bradley A Hamilton1, J Fraser Wright1
1Center for Definitive and Curative Medicine, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, United States.
Frontiers in Immunology
|June 4, 2021
Summary
Host immune responses hinder adeno-associated virus (AAV) gene therapies. Removing pathogen patterns from AAV genomes can prevent immune danger signals, overcoming these barriers for successful gene therapy development.
Area of Science:
- Immunology
- Gene Therapy
- Virology
Background:
- Host immune responses are a significant obstacle to the widespread success of adeno-associated virus (AAV)-based gene therapies.
- Inflammation and limited durable gene expression caused by immune reactions impede therapeutic efficacy.
Purpose of the Study:
- To discuss the mechanisms of humoral and cellular immune responses against AAV vectors.
- To propose a strategy for overcoming immunological barriers in AAV gene therapy.
Main Methods:
- Review and discussion of existing literature on immune responses to AAV vectors.
- Analysis of molecular patterns within AAV vector genomes.
Main Results:
- Humoral and cellular immune responses target AAV vectors, limiting gene therapy effectiveness.
- Pathogen-associated molecular patterns (PAMPs) in vector genomes can trigger innate immune responses.
Conclusions:
- Removing PAMPs from AAV vector genomes is a crucial strategy to prevent innate immune danger signals.
- This approach can help overcome immunological barriers, enabling more successful AAV-based gene therapies.
