Gene Therapy in Spinal Muscular Atrophy (SMA) Models Using Intracerebroventricular Injection into Neonatal Mice

  • Monir ShababiEmail author
  • Erkan Y. Osman
  • Christian L. Lorson
Part of the Neuromethods book series (NM, volume 98)


Successful gene therapy for neurodegenerative disorders in clinical trials depends upon the success of the gene therapy applications in preclinical models of the disease. Preclinical animal studies often proceed to larger animal models and are subsequently incorporated into the final design of the clinical trials. Recent gene therapy advancements with preclinical animal models of spinal muscular atrophy (SMA) have made the move from the bench research to an actual treatment a more achievable reality. In this chapter, we gathered the most recent gene therapy advancements in SMA animal models and discuss the possibility of gene therapy clinical trials. We will also discuss the outcome of our gene therapy approaches in the transduced tissues of SMA mice using single-stranded and self-complementary AAV vectors. One method of the transgene delivery into the central nervous system (CNS) is intracerebroventricular (ICV) injection. In this chapter, we provide a detailed protocol of the ICV injection into the murine brain at early postnatal time points with photographs demonstrating each step of the process.

Key words

Gene therapy AAV vectors SMN SMA ICV injection 



We like to thank John Marston for the maintenance of the mouse colony and Katie Robinson for her assistance in harvesting the organs. This work was supported by grants from MDA and SMA Europe (M.S.) and Fight SMA (C.L.L.).


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Copyright information

© Springer Science+Business Media New York 2015

Authors and Affiliations

  • Monir Shababi
    • 1
    Email author
  • Erkan Y. Osman
    • 2
  • Christian L. Lorson
    • 1
    • 2
  1. 1.Department of Veterinary PathobiologyUniversity of MissouriColumbiaUSA
  2. 2.Department of Molecular Microbiology and ImmunologySchool of MedicineColumbiaUSA

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