Aspartoacylase mRNA (ASPA mRNA)

Aspartoacylase (ASPA) mRNA is the transcript of the ASPA gene, which encodes the enzyme responsible for breaking down N-acetylaspartate (NAA) into aspartate and acetate in the brain. This process is critical for maintaining brain health, as acetate is a necessary precursor for the synthesis of myelin, the protective sheath around nerve fibers. In Canavan disease, mutations in the ASPA gene lead to a deficiency of the functional enzyme, resulting in the toxic accumulation of NAA and a lack of acetate, which causes progressive destruction of white matter (leukodystrophy). Therapeutic strategies targeting ASPA mRNA involve delivering functional copies of the mRNA or the gene itself to the central nervous system to restore enzyme activity. Current clinical efforts, such as gene therapies using adeno-associated virus (AAV) vectors, aim to provide long-term expression of ASPA mRNA in oligodendrocytes to halt or reverse the neurodegeneration associated with the disease.

Other names

Aminoacylase-2 mRNAN-acetylaspartate amidohydrolase mRNAACY2 mRNA

mRNA replacement or gene therapy to restore functional aspartoacylase enzyme activity, thereby reducing toxic N-acetylaspartate (NAA) levels and providing acetate for myelin synthesis.