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M19 Sertoli Cell Transplant: New Hope for the Treatment of Huntington Disease (HD)
  1. A Di Pardo1,
  2. G Luca2,
  3. R Calafiore3,
  4. V Maglione1,
  5. M Favellato1,
  6. E Amico1,
  7. A Minelli2,
  8. F Squitieri1
  1. 1Neurogenetics and Rare Disease Centre, IRCCS Neuromed, Pozzilli, IT
  2. 2Department of Experimental Medicine and Biochemical Sciences, Faculty of Medicine and Surgery, Università di Perugia
  3. 3Department of Medicine, Faculty of Medicine and Surgery, Università di Perugia

Abstract

Introduction Huntington disease (HD) is a neurodegenerative disorder characterised by progressive worsening of behavioural, cognitive and motor function. The disease-causing mutation is an expanded CAG repeat in the huntigtin gene. Although rapid advancing basic and translational research has identified numerous potential targets for treating HD, no definitive cure is currently available. Promising research on alternative therapeutic approaches, however, offers new hope. Experimental evidence indicates that testis-derived Sertoli cells (SCs), which have been demonstrated to play a critical role in different pathophysiological activities, have the potential to be a very useful tool for chronic neurodegenerative conditions.

Objective To test the ability of SCs to secrete molecules with neuroprotective properties and whether their transplantation into HD R6/2 mice may represent an effective strategy for developing therapies.

Methods In vitroexperiments were carried out in mouse striatal-derived cells with 111 CAG repeat (STHdh111/111), cultured in apoptotic conditions, in presence or absence of conditioned medium from SCa. Cell survival was evaluated by Annexin V staining. In vivoexperiments were performed on transgenic R6/2 mice and wild type littermates. The effect of SCs trasplantation on motor function and longevity was assessed by behavioural testing and survival analysis, respectively.

Results Coherently with in vitrostudies that highlighted protection of cells from apoptosis, in vivostudies showed that SCs transplatation efficiently ameliorated the overall motor function in R6/2 mice and significantly prolonged lifespan in the same mice.

Conclusions The beneficial effects of SCs trasplantation in HD models provide new perspectives for innovative therapies in HD, however further studies are warranted.

KeyWords
  • HD
  • R6/2
  • Scs transplantation

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