New therapies on the horizon: Targeted protein degradation in neuroscience

JA Gregory, CM Hickey, J Chavez, AM Cacace - Cell Chemical Biology, 2024 - cell.com
This minireview explores the burgeoning field of targeted protein degradation (TPD) and its
promising applications in neuroscience and clinical development. TPD offers innovative …

Deep learning facilitates efficient optimization of antisense oligonucleotide drugs

S Lin, L Hong, DQ Wei, Y **ong - Molecular Therapy-Nucleic Acids, 2024 - cell.com
Nucleic acid therapeutics exert their therapeutic effects by targeting specific genes through
complementary sequence recognition to inhibit the expression of certain proteins or facilitate …

[HTML][HTML] Convergence on CaMK4: a key modulator of autism-associated signaling pathways in neurons

J Kaiser, A Risteska, AG Muller, H Sun, B Lei, K Nay… - Biological …, 2024 - Elsevier
Although the precise underlying cause (s) of autism spectrum disorder remain unclear, more
than 1000 rare genetic variations are associated with the condition. For a large number of …

Male proband with intractable seizures and a de novo start codon disrupting variant in GLUL

E Carbonell, SL Stenton, VS Ganesh, J Ma… - Human Genetics and …, 2025 - cell.com
Biallelic variants in GLUL, encoding glutamine synthetase and responsible for the
conversion of glutamate to glutamine, are associated with a severe recessive disease due to …

[PDF][PDF] Streamlining Drug Repurposing: Optimizing Candidate Prioritization to Facilitate Clinical Adoption and Minimize Attrition Rates

E Jones, S Taluri, E Wilk, B Lasseigne - 2024 - preprints.org
Drug repurposing, identifying FDA-approved drugs for new indications, reduces the time and
money required for a drug to reach the clinic and capitalizes on the increasing number of …