Targeted intraspinal injections to assess therapies in rodent models of neurological disorders

Nat Protoc. 2019 Feb;14(2):331-349. doi: 10.1038/s41596-018-0095-5.

Abstract

Despite decades of research, pharmacological therapies for spinal cord motor pathologies are limited. Alternatives using macromolecular, viral, or cell-based therapies show early promise. However, introducing these substances into the spinal cord, past the blood-brain barrier, without causing injury is challenging. We describe a technique for intraspinal injection targeting the lumbar ventral horn in rodents. This technique preserves motor performance and has a proven track record of translation into phase 1 and 2 clinical trials in amyotrophic lateral sclerosis (ALS) patients. The procedure, in brief, involves exposure of the thoracolumbar spine and dissection of paraspinous muscles over the target vertebrae. Following laminectomy, the spine is affixed to a stereotactic frame, permitting precise and reproducible injection throughout the lumbar spine. We have used this protocol to inject various stem cell types, primarily human spinal stem cells (HSSCs); however, the injection is adaptable to any candidate therapeutic cell, virus, or macromolecule product. In addition to a detailed procedure, we provide stereotactic coordinates that assist in targeting of the lumbar spine and instructional videos. The protocol takes ~2 h per animal.

Publication types

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

MeSH terms

  • Amyotrophic Lateral Sclerosis / pathology
  • Amyotrophic Lateral Sclerosis / surgery*
  • Amyotrophic Lateral Sclerosis / therapy
  • Animals
  • Disease Models, Animal
  • Dissection / methods*
  • Humans
  • Injections, Spinal / methods*
  • Lumbosacral Region / surgery
  • Male
  • Mice, Transgenic
  • Motor Activity / physiology
  • Paraspinal Muscles / surgery
  • Rotarod Performance Test
  • Spinal Cord / pathology
  • Spinal Cord / surgery*
  • Stem Cell Transplantation / methods
  • Stereotaxic Techniques*
  • Transplantation, Heterologous