Cerebellomedullary Cistern Delivery for AAV-Based Gene Therapy: A Technical Note for Nonhuman Primates

Hum Gene Ther Methods. 2016 Feb;27(1):13-6. doi: 10.1089/hgtb.2015.129.

Abstract

Accessing cerebrospinal fluid (CSF) from the craniocervical junction through the posterior atlanto-occipital membrane via cerebellomedullary injection (also known as cisternal puncture or cisterna magna injection) has become a standard procedure in preclinical studies. Such delivery provides broader coverage to the central and peripheral nervous system unlike local parenchymal delivery alone. As a clinical application, this approach offers a more reliable method for neurological gene replacement delivery in infants, where skull-mounted devices are not indicated. Here we describe a consistent, precise, and safe method for CSF injection with minimal equipment and technical skills.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Central Nervous System / metabolism*
  • Cisterna Magna
  • Dependovirus / genetics*
  • Female
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Genetic Vectors / administration & dosage*
  • Genetic Vectors / genetics*
  • Humans
  • Injections, Epidural
  • Male
  • Primates