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Updated: May 5, 2026

Intracranial Injection of Adeno-associated Viral Vectors
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シリアンハムスターに頭蓋内注射した後のVCN-01の毒性および生物分布

Daniel Palacios-Alonso1,2,3, Miriam Bazan-Peregrino4, Marta Zalacain1,2,3

  • 1Department of Pediatrics, Clinica Universidad de Navarra, Pamplona, Spain.

Human gene therapy
|August 29, 2025
PubMed
まとめ

腫瘍性アデノウイルスであるVCN-01の頭蓋内投与は,臨床前の脳腫瘍モデルで好ましい安全性プロファイルを示しています. バイオディストリビューションの研究は,VCN-01が脳に限られていることを確認し,小児脳腫瘍の治療の可能性を裏付けています.

キーワード:
VCN-01 について生物学的分布頭蓋内投与腫瘍性アデノウイルス小児脳腫瘍毒性について

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Toxicity Testing in Animals

Toxicity tests in animals are grounded on two main assumptions: first, the effects observed in laboratory animals can be extrapolated to humans, especially when adjusted for body surface area; second, high-dose exposure in animals is essential to identify potential human hazards from lower doses. This is based on the quantal dose-response concept, which faces the challenge of extrapolating results from relatively few test animals to much larger human populations. For example, a 0.01% incidence...
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