Detailed method for intrathecal delivery of gene therapeutics by direct lumbar puncture in mice

Research output: Chapter in Book/Report/Conference proceedingChapter

7 Scopus citations


Delivery of viral vectors directly into the central nervous system (CNS) has emerged as an important tool for the refinement of gene therapy. Intrathecal delivery by direct lumbar puncture in conscious rodents offers a minimally invasive approach that avoids tissue damage and/or destruction. Here we describe delivery of small quantities of viral vector product to the intrathecal space of rodents via direct lumbar puncture aided by a catheter.

Original languageEnglish (US)
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Number of pages8
StatePublished - 2019

Publication series

NameMethods in molecular biology (Clifton, N.J.)
PublisherHumana Press
ISSN (Print)1064-3745

Bibliographical note

Funding Information:
This work was supported by NIDA grant R01DA035931-01, DoD grant W81XWH-15-1-0494, T32 training grant T32DA07234 to K.P., and T32 training grant T32DA007097 to C.P.


  • AAV gene therapy
  • Direct lumbar puncture
  • Gene therapy
  • Intrathecal
  • Intrathecal gene delivery
  • Injections, Spinal
  • Genetic Therapy
  • Humans
  • Catheters
  • Dependovirus/genetics
  • Animals
  • Genetic Vectors/administration & dosage
  • Female
  • Mice
  • Spinal Puncture/instrumentation

PubMed: MeSH publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Journal Article
  • Research Support, N.I.H., Extramural


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