Highthroughput Screening and Cell Repository Core


M. Carrie Miceli, Ph.D.


The importance of high throughput screening (HTS) in an academic environment is evidenced by inclusion of Molecular Library screening as one of five major points in the current NIH Roadmap for Medical Research. At UCLA, The Molecular Screening Shared Resource (MSSR) was established in 2003 to provide the whole UCLA community with HTS technology that would be otherwise inaccessible to individual researchers. This shared resource provides robotic-based HTS capabilities for academic researchers who wish to pursue research in chemical- and functional-genomics using chemical library and siRNA library screens and is one of only 36 academic high throughput screening centers world-wide (one of 31 in the United States) as identified in a recent review 1.

Core B of the Center for Duchenne Muscular Dystrophy at UCLA represents an exciting new resource, which will make a significant impact on research at UCLA in muscle biology. Investigators within the Center for DMD at UCLA have capitalized on the presence of the MSSR by creating Core B, which provides cell models, expertise and space for cell expansion and handling of muscle lineage and muscular dystrophy relevant cells. This muscle-relevant HTS Core utilizes the robotics and imaging capabilities of the MSSR and provides expertise, space, resources, and guidance on muscle cell and fibroblast culture. In addition, the core provides muscle and fibroblast cell lines with specific muscular dystrophy mutations and serves as a repository for developed assays that might serve as useful secondary assays in future screens.


The HTS screening core is available to advise core users in assay development and provide access to available muscle and muscular dystrophy relevant cell models. In addition, the Core will make the following reagents available to researchers interested in utilizing or further developing these models.

  • C2C12 mouse myoblast (capable of fusing into myotubes in culture).
  • H2-Kb-tsA58 mdx cells (mdx myoblast expressing temperature sensitive Sv40 regulated growth and fusion)
  • Human dystrophin exon 50 skipping reporter expressed in C2C12 cells (Ex50GFP) (with permission from Qi Lu) (described in Figure 1)
  • Human dystrophin exon 45 Ex45GFP/RFPpFLARE reporter expression in C2C12 (in development, the advantages and features of pFLARE exon skipping vector, including ability to distinguish true skippers from enhancers of generalize translation and transcription is well articulated in the pilot and feasibility project # 2 as it was developed in the Black lab)

*DMD patient derived human dystrophic fibroblast inducible to myoblast/muscle lineage (in development)
*DMD patient derived iPSC inducible/selectable to myoblast/muscle lineage (in development)

  • Stable C2C12 cells transfected with cDNA encoding the CT GalNAc transferase enzyme that adds GalNAc residues to the unique O-mannosyl glycans on dystroglycan.
  • Primary mouse muscle derived myoblasts from mdx and wildtype mice.

Other services:

  • Cell culture expansion and plating (in HTS format) and MSSR interfacing services.
  • Retrieval and analysis of data collected at the MSSR and data mining.
  • Aid in development and implementation of secondary assays for lead hit compound validation.


The Core Center is dedicated to understanding processes in degenerative muscle disorders and to identifying therapeutic interventions. In order to most rapidly effect gains, the Center provides a host of interactive intellectual, administrative and research cores. The Center is comprised of four core facilities, all designed to facilitate discovery of therapeutics and test hypotheses related to muscle research. These cores include:

Highthroughput Screening and Cell Repository Muscle Phenotyping and Imaging Bioinformatics and Genomics Back

A primary goal for the Center in creating these Cores is to make available generically useful resources that can be distributed to other researchers in muscular dystrophy or to basic scientists interested in muscle research. The Center is structured to enrich existing muscle research programs on campus by providing its members with access to unique core facilities, pilot funding mechanisms, seminars and an annual retreat focused on muscle research.

Furthermore, the Core Center brings together investigators with a variety of specific interests, some of which are not directly muscle related, to benefit research in muscle disorders.