Drugs Discovery Assets:
In Vitro Tools
Drugs Discovery Assets: In Vitro Tools
In vitro assays are performed on tissues or cells maintained outside of the body, usually in the form of a cell culture plate or test tube. In vitro assays are important because they provide the opportunity to test compounds and investigate SMA biology in human and animal model cells. These tools also provide convenient and rapid readouts for high-throughput screening of compounds. The SMA Foundation has invested close to $13M in establishing a panel of in vitro platforms and tools in collaboration with researchers for testing SMA therapeutics and elucidating SMA biology. The majority of these assays were developed at the Columbia University Motor Neuron Center (MNC) and the Harvard Stem Cell Institute (HSCI). The complete list of SMA Foundation in vitro tools is available below. For more information, please contact us at [email protected].
| Research Tool | Readouts | Institution | SMN/Downstream |
| SMA ELISA | SMN Protein | Enzo Life Sciences/SMAF | SMN |
| Inducible knockdown of mSMN in mouse fibroblasts | Proliferation | MNC (Livio Pellizzoni) | Downstream |
| Inducible knockdown of mSMN+SMN2 in mouse fibroblasts | ProliferationSMN protein level/function | MNC (Livio Pellizzoni) | Both |
| ES-derived motor neurons from SMA mice | Survival of motor neurons SMN protein level/function | MNC (Hynek Wichterle), HSCI (Lee Rubin) | Both |
| SMA patient PGD-derived human motor neurons | Survival of motor neuronsSMN protein level/function | HSCI (Kevin Eggan) | Both |
| Inducible knockdown of mSMN in ES-derived mouse motor neurons | Survival of motor neurons | MNC (Livio Pellizzoni) | Downstream |
| Knockdown of mSMN in ES-derived mouse motor neurons | Survival of motor neurons | HSCI (Lee Rubin) | Downstream |
| Myoblasts from SMA mice | SMN protein level/function | NYU (Steve Burden) | SMN |
| Co-culture myoblasts with ES-derived motor neurons from SMA mice | Synapse Formation | HSCI (Lee Rubin) | Downstream |
| Mouse motor neurons and SMA patient fibroblasts | SMN Protein | HSCI (Lee Rubin) | SMN |
Table 1. In vitro assays available for SMA drug development. Assays are listed with descriptions of their readouts, their developers, and institutions. The table also details whether individual assays provide information directly on SMN levels or functions, on other metrics that are downstream of SMN activity, or on both direct and downstream SMN readouts.