Neural Regeneration Research ›› 2023, Vol. 18 ›› Issue (12): 2663-2664.doi: 10.4103/1673-5374.373658

Previous Articles     Next Articles

Recent advances in RNA-targeting therapy for neurological diseases

Satheesh Kumar, Guei-Sheung Liu*   

  1. Center for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, VIC, Australia (Kumar S, Liu GS)
    Menzies Institute for Medical Research, University of Tasmania, Hobart, TAS, Australia (Kumar S, Liu GS) 
    Ophthalmology, Department of Surgery, University of Melbourne, East Melbourne, VIC, Australia (Kumar S, Liu GS)
    Aier Eye Institute, Changsha, Hunan Province, China (Liu GS) 
  • Online:2023-12-15 Published:2023-06-14
  • Contact: Guei-Sheung Liu, PhD, rickliu0817@gmail.com.
  • Supported by:
    This work was funded by grants from the National Health and Medical Research Council of Australia (1185600; to GSL) and Retina Australia (to GSL). The Center for Eye Research Australia receives Operational Infrastructure Support from the Victorian Government.

PDF

77

Abstract: Advances in sequencing and molecular technology now allow us to understand the genetic underpinnings of complex diseases such as neurological disorders. Genetic variations (or mutations) in the DNA sequence of single genes have been implicated in neurological diseases such as Huntington’s disease and spinal muscular atrophy. As a result, the development of gene therapies for neurological diseases is now a feasible endeavor. Indeed, gene therapy for neurological diseases has recently been invigorated by the market approvals of Zolgensma® (onasemnogene abeparvovec) in 2019 and UpstazaTM (eladocagene exuparvovec) in 2022. These gene therapies deliver a transgene to compensate for an aberrant or missing gene for the therapeutic benefit of neurological diseases and have demonstrated significant clinical potential. However, current gene therapy is limited to loss-of-function genetic diseases, and the delivery approach for large genes has been a challenge.