TAR DNA-binding protein of 43 kDa (TDP-43) and amyotrophic lateral sclerosis (ALS): a promising therapeutic target.
Yara Al OjaimiAudrey DangoumauHugo AlarcanRudolf HergesheimerPatrick Vourc'hPhilippe CorciaDébora LanznasterHélène BlascoPublished in: Expert opinion on therapeutic targets (2022)
The complexity of ALS and TDP-43 proteinopathy generates challenges for the development of novel therapeutic approaches. However, the critical involvement of TDP-43 in the initiation and progression of ALS, makes it a promising therapeutic target. Further research should be centered on the development of precision strategies, consideration of patient subgroups, the prevention of the mislocalization of TDP-43 and restoration of the lost functions of TPD-43. .