Pulling Back the Curtain: Combinatorial Screening
What are the realities of translating exciting cell and gene therapy research into something more tangible? And what solutions are being developed as a result?
The regenerative medicine field is relatively new – where do you see its potential?
Whereas traditional pharmaceutical and biotechnology companies have focused on using small molecules and proteins, respectively, to target specific molecular pathways within cells, regenerative medicine aims to develop cures for diseases rather than manage symptoms. For example, pluripotent stem cells may be able to regenerate retinal pigmented epithelial cells lost during age-related macular degeneration or dopaminergic neurons destroyed by Parkinson’s disease. Regenerative medicine can also improve the performance of immune cells unable to cope with aggressive diseases, such as certain cancers. A recent, highly publicised example has been the unprecedented efficacy of CAR-T therapies in fighting blood cancers, such as acute lymphoblastic leukemia. Furthermore, where a disease is clearly defined as being caused by a specific genetic defect, novel gene-editing technologies are able to permanently correct these defects either ex vivo or directly within the patients themselves.
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