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Brain Sciences

Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases

Brain Sciences, 2020 · Cited by 114 papers · doi:10.3390/brainsci10020119

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What this article backs

• Gene Therapy ⚡ Neuroinflammation
• Neuroinflammation ↓ Transgene Expression
• Cns Targeted Aav Delivery ⚡ Neuroinflammation
• Cns Targeted Aav Delivery ↑ Transgene Expression
• Systemic Aav Dose ↑ Neuroinflammation

• Gene Therapy triggers Neuroinflammation.
• Neuroinflammation decreases Transgene Expression.
• Cns Targeted Aav Delivery triggers Neuroinflammation.
• Cns Targeted Aav Delivery increases Transgene Expression.
• Systemic Aav Dose increases Neuroinflammation.

In its own words

neuroinflammatory responses after CNS-targeted delivery of AAV are a critical concern. Reported signs of AAV-associated neuroinflammation in preclinical studies include dorsal root ganglion (DRG) and spinal cord pathology with mononuclear cell infiltration.

Large doses of systemically administered AAV stimulate host immune responses, resulting in anti-capsid and anti-transgene immunity with implications for transgene expression, treatment longevity, and patient safety.

Delivering lower doses directly to the central nervous system (CNS) is a promising alternative, resulting in higher transgene expression with decreased immune responses.

resulting in anti-capsid and anti-transgene immunity with implications for transgene expression, treatment longevity, and patient safety.

Large doses of systemically administered AAV stimulate host immune responses, resulting in anti-capsid and anti-transgene immunity