research and DEVELOPMENT

PRECISION™ Discovery

CELLTIUM has developed PRECISION™, a state-of-the-art drug discovery platform implemented with high-throughput and high-content automated workflows. Our IRIS™ (intuitive reconn imaging system) and machine learning framework further drive our CMT BigData and A.I. architecture. Revolutionized by innovative modular technologies, our PRECISION™ platform targets Charcot-Marie-Tooth disease (CMT), diabetic peripheral neuropathy (DPN), and chemotherapy-induced peripheral neuropathy (CIPN).


CELLTIUM is testing customizable proof-of-concept for gene therapy targeting Charcot-Marie-Tooth diseases. We refine adeno-associated virus (AAV) as an impactful gene transfer system. CELLTIUM completes CRISPR (clustered regularly interspaced short palindromic repeats) techniques to repair disease-causing genetic defects and ultimately restore the normal functions of target cells.


Charcot-Marie-Tooth Disease

Schwann cell is the main non-neuronal cell-type, known as glia or glial cells, in the peripheral nervous system. Schwann cell forms the lipid-rich membranous myelin sheath surrounding peripheral axons, which functions as electrical insulator to ensure proper propagation of neural information along peripheral nerves. In addition, Schwann cell maintains environmental and metabolic homeostasis for peripheral nerves. Many genetic mutations found in Charcot-Marie-Tooth patients primarily interfere with Schwann cell development and function including myelin sheath formation and maintenance, while neuronal or axonal defects are manifested in CMT2 subtypes.
Schwann cell, Myelin sheath, Axon, Neuron. Image Credit: Designua / Shutterstock

Research Articles

Viader A, Sasaki Y, Kim S, Strickland A, Workman CS, Yang K, Gross RW, Milbrandt J. Aberrant Schwann cell lipid metabolism linked to mitochondrial deficits leads to axon degeneration and neuropathy. Neuron. 2013 Mar 6;77(5):886-98. doi: 10.1016/j.neuron.2013.01.012. PubMed PMID: 23473319; PubMed Central PMCID: PMC3594792.

Redmann V, Lamb CA, Hwang S, Orchard RC, Kim S, Razi M, Milam A, Park S, Yokoyama CC, Kambal A, Kreamalmeyer D, Bosch MK, Xiao M, Green K, Kim J, Pruett-Miller SM, Ornitz DM, Allen PM, Beatty WL, Schmidt RE, DiAntonio A, Tooze SA, Virgin HW. Clec16a is Critical for Autolysosome Function and Purkinje Cell Survival. Sci Rep. 2016 Mar 18;6:23326. doi: 10.1038/srep23326. PubMed PMID: 26987296; PubMed Central PMCID: PMC4796910.

Kim S, Maynard JC, Sasaki Y, Strickland A, Sherman DL, Brophy PJ, Burlingame AL, Milbrandt J. Schwann Cell O-GlcNAc Glycosylation Is Required for Myelin Maintenance and Axon Integrity. J Neurosci. 2016 Sep 14;36(37):9633-46. doi: 10.1523/JNEUROSCI.1235-16.2016. PubMed PMID: 27629714; PubMed Central PMCID: PMC5039245.

Sasaki Y, Hackett AR, Kim S, Strickland A, Milbrandt J. Dysregulation of NAD+ Metabolism Induces a Schwann Cell Dedifferentiation Program. J Neurosci. 2018 Jul 18;38(29):6546-6562. doi: 10.1523/JNEUROSCI.3304-17.2018. Epub 2018 Jun 19. PubMed PMID: 29921717; PubMed Central PMCID: PMC6052240.

Kim S, Maynard JC, Strickland A, Burlingame AL, Milbrandt J. Schwann cell O-GlcNAcylation promotes peripheral nerve remyelination via attenuation of the AP-1 transcription factor JUN. Proc Natl Acad Sci U S A. 2018 Jul 31;115(31):8019-8024. doi: 10.1073/pnas.1805538115. Epub 2018 Jul 16. PubMed PMID: 30012597; PubMed Central PMCID: PMC6077742.

Jang HS, Shah NM, Du AY, Dailey ZZ, Pehrsson EC, Godoy PM, Zhang D, Li D, Xing X, Kim S, O’Donnell D, Gordon JI, Wang T. Transposable elements drive widespread expression of oncogenes in human cancers. Nat Genet. 2019 Apr;51(4):611-617. doi: 10.1038/s41588-019-0373-3. Epub 2019 Mar 29. PubMed PMID: 30926969; PubMed Central PMCID: PMC6443099.