This could explain some of the findings for example in the recently halted ASO trial in Huntington's disease.105,106 Thus, while the silencing of mHTT will certainly have beneficial consequences, as demonstrated in our study by efficiently lowering LAMP1 in the neurites, the silencing of wtHTT will also come with loss-of-function consequences on similar cellular pathways. This evidence concerns the gene LAMP1 and Huntington disease.