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Output Catalog
ASAP is committed to accelerating the pace of discovery and informing a path to a cure for Parkinson’s disease through collaboration, research-enabling resources, and data sharing. We’ve created this catalog to showcase the research outputs and tools developed by ASAP-funded programs.
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Delivery of RNA Therapeutics: The Great Endosomal Escape!
RNA therapeutics show promise in treating various diseases by targeting specific genes without needing prior protein knowledge. Overcoming cellular defenses like endosomal entrapment is crucial for their widespread use in treating diseases.
Brain Repair by Cell Replacement via In Situ Neuronal Reprogramming
Neurodegenerative diseases, characterized by progressive neural loss, have been some of the most challenging medical problems in aging societies. Treatment strategies such as symptom management have little impact on disease progression, while…
Endosomal escape of RNA therapeutics: How do we solve this rate-limiting problem?
With over 15 FDA approved drugs on the market and numerous ongoing clinical trials, RNA therapeutics, such as small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs), have shown great potential to treat human disease. Their mechanism…
Therapeutic Potential of PTB Inhibition Through Converting Glial Cells to Neurons in the Brain
Cell replacement therapy represents a promising approach for treating neurodegenerative diseases. Contrary to the common addition strategy to generate new neurons from glia by overexpressing a lineage-specific transcription factor(s), a recent study…
RNAseq FASTQ: Effect of age and trisomy on transcriptional profile in cortex of Dp16 mice
Cortex was isolated from control and Dp16 mice at 5 months of age and 14-16 months of age, and RNA was isolated and sequenced (5 mice per group, for a total of 20 mice). The sample IDs are as follows: AJ1-AJ5: 2N young AJ6-AJ10 2N old AJ11-AJ15 Dp16…