GEN Exclusives

More »

GEN News Highlights

More »
Feb 3, 2011

Cedars-Sinai Team Wins $1.9M to Develop iPSC System for Testing Compounds against SMA

  • A research team at the Cedars-Sinai Regenerative Medicine Institute has been awarded a $1.9 million grant by the California institute for Regenerative Medicine. It will fund development of an induced pluripotent stem cell-based platform that will help researchers identify potential new drug candidates for spinal muscular atrophy (SMA).

    SMA is caused by a genetic mutation in the SMN1 gene, which stops motor neurons manufacturing the critical protein SMN. Another gene, SMN2, does manufacture the SMN protein, but not enough is produced overall to compensate for the SMN1 gene-related deficit, the Sinai researchers explain.

    Their team, led by Druv Sareen, Ph.D., aims to generate pluripotent stem cells from the skin cells of SMA patients and then trigger these to develop into motor neurons with the mutated SMN1 gene and normal SMN2 gene. This platform will then be used to test compounds for their ability to increase production of SMN by the functioning SMN2 gene.

    “Pharmaceutical and biotech companies have comprehensive libraries of chemically diverse compounds, some of which could potentially be developed into drug treatments to help SMA patients,” Dr. Sareen remarks. “We will be able to test these drug libraries relatively quickly using our high-content screening approach. We hope to identify novel compounds that target the SMN2 gene specifically in patient motor neurons."

Add a comment

  • You must be signed in to perform this action.
    Click here to Login or Register for free.
    You will be taken back to your selected item after Login/Registration.

Related content


GEN Jobs powered by connects you directly to employers in pharma, biotech, and the life sciences. View 40 to 50 fresh job postings daily or search for employment opportunities including those in R&D, clinical research, QA/QC, biomanufacturing, and regulatory affairs.
More »

Be sure to take the GEN Poll

Patient Access to Genetic Information

Do you think patients have the absolute right to gain access to their own genetic information from medical or clinical laboratories?

More »