Determination and investigation of the therapeutic targets of HDAC6 inhibition in amyotrophic lateral sclerosis (ALS) KU Leuven
Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disease characterized by the selective death of motor neurons leading to complete paralysis and death of the patient on average 2 to 5 years after the diagnosis. We discovered that inhibition of histone deacetylase 6 (HDAC6) counteracts diseaserelated phenotypes in iPSC-derived motor neurons containing mutations in the genes encoding FUS and TDP-43. HDAC6 is a cytoplasmic ...