| abstract |
The present invention relates to a polynucleotide comprising a GBA nucleotide sequence encoding a GCase protein or a fragment thereof, wherein a part of the coding sequence is not wild-type. The present invention also relates to viral particles comprising a recombinant genome comprising a polynucleotide of the present invention, polynucleotides or compositions comprising the viral particles, and methods and uses of the polynucleotides, viral particles or compositions. |