Publications

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Evaluating the analytical validity of circulating tumor DNA sequencing assays for precision oncology

Circulating tumor DNA (ctDNA) sequencing is being rapidly adopted in precision oncology, but the accuracy, sensitivity and reproducibility of ctDNA assays is poorly understood. Here we report the findings of…

Journal: Nature Biotechnology


pS421 huntingtin modulates mitochondrial phenotypes and confers neuroprotection in an HD hiPSC model

Huntington disease (HD), a devastating hereditary neurodegenerative disorder, is caused by an expansion of a CAG trinucleotide repeat that encodes a polyglutamine (polyQ) tract in the huntingtin (HTT) gene.

Journal: Cell Death & Disease


Advanced bioinformatics rapidly identifies existing therapeutics for patients with coronavirus disease-2019 (COVID-19)

The recent global pandemic has placed a high priority on identifying drugs to prevent or lessen clinical infection of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), caused by Coronavirus disease-2019…

Journal: Journal of Translational Medicine


Deep immune profiling of patients treated with lenalidomide and dexamethasone with or without daratumumab

CD38-targeted antibody, daratumumab, is approved for the treatment of multiple myeloma (MM). Phase 1/2 studies GEN501/SIRIUS revealed a novel immunomodulatory mechanism of action (MOA) of daratumumab that enhanced the immune…

Journal: Leukemia


Large-scale transcriptomic analysis reveals that pridopidine reverses aberrant gene expression and activates neuroprotective pathways in the YAC128 HD mouse

Huntington Disease (HD) is an incurable autosomal dominant neurodegenerative disorder driven by an expansion repeat giving rise to the mutant huntingtin protein (mHtt), which is known to disrupt a multitude…

Journal: Molecular Neurodegeneration


Integrity, standards, and QC-related issues with big-data in pre-clinical drug discovery

The tremendous expansion of data analytics and public and private big datasets presents an important opportunity for pre-clinical drug discovery and development. In the field of life sciences, the growth…

Journal: Biochemical Pharmacology


The international MAQC Society launches to enhance reproducibility of high-throughput technologies

Reproducibility is a fundamental hallmark of good science. The US Food and Drug Administration (FDA)-led Microarray and Sequencing Quality Control (MAQC/SEQC) consortia conducted three projects 1,2,3 to assess the reliability and…

Journal: Nature Biotechnology 


Early pridopidine treatment improves behavioral and transcriptional deficits in YAC128 Huntington disease mice

Pridopidine is currently under clinical development for Huntington disease (HD), with on-going studies to better characterize its therapeutic benefit and mode of action. Pridopidine was administered either prior to the…

Journal: JCI Insight


Compositional differences between Copaxone and Glatopa are reflected in altered immunomodulation ex vivo in a mouse model.

Copaxone (glatiramer acetate, GA), a structurally and compositionally complex polypeptide nonbiological drug, is an effective treatment for multiple sclerosis, with a well-established favorable safety profile. The short antigenic polypeptide sequences…

Journal: Annals of the New York Academy of Sciences


Drug repurposing from the perspective of pharmaceutical companies

Drug repurposing holds the potential to bring medications with known safety profiles to new patient populations. Numerous examples exist for the identification of new indications for existing molecules, most stemming…

Journal: British Journal of Pharmacology


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