Recent advances in RNA-targeting therapy for neurological diseases

<p> </p> <p>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 Upstaza TM (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</p>