Activity-dependent neuroprotective protein mRNA (ADNP mRNA) (ADNP mRNA)

Activity-dependent neuroprotective protein (ADNP) mRNA and pre-mRNA are critical therapeutic targets for treating ADNP syndrome (Helsmoortel-Van der Aa syndrome), a rare neurodevelopmental disorder (MedlinePlus, 2017). The ADNP gene encodes a protein essential for brain development and chromatin remodeling through its interaction with the SWI/SNF complex (Wikipedia, 2024). In ADNP syndrome, haploinsufficiency occurs due to de novo mutations, leading to insufficient protein levels (University of California, 2022). Therapeutic strategies targeting the mRNA include antisense oligonucleotides (ASOs) designed to enhance translation efficiency by blocking inhibitory upstream open reading frames (uORFs) (University of California, 2022). These ASOs aim to increase the production of functional protein from the remaining healthy allele to compensate for the mutation (WIPO, 2025). Beyond ADNP syndrome, dysregulation of ADNP mRNA has been implicated in Alzheimer's disease and schizophrenia, where levels are often found to be decreased or altered (Frontiers in Aging Neuroscience, 2014). ADNP is also overexpressed in certain cancers, such as colorectal cancer, where it may promote tumor progression through the WNT signaling pathway (AACR, 2015). The target is particularly attractive for precision medicine approaches in autism spectrum disorders with known genetic origins (University of California, 2022). Challenges in targeting ADNP mRNA include ensuring effective delivery to the central nervous system and minimizing off-target hybridization (NIH, 2025). Overall, ADNP mRNA represents a high-potential target for both restoring function in genetic deficiency and providing neuroprotection in broader pathologies.

Other names

Activity-dependent neuroprotector homeobox mRNAADNP1 mRNAADNP pre-mRNA

Translation enhancement via inhibition of upstream open reading frames (uORFs); Splice modulation to increase functional protein expression.