Manasa Prahlad

2020
Biochemistry
Description: 
I am currently working in the lab of Dr. Lawrence Chasin, in the Biology department of Columbia University. Dr. Chasin’s lab specializes in the splicing mechanism by which the primary RNA transcript becomes mRNA. Our current project involves applying this research towards the treatment of protein related disorders such as Duchenne muscular dystrophy (DMD). More specifically, we intend to do this by promoting the skipping of certain exons during the splicing of the gene’s pre-mRNA transcript. In the example of DMD, skipping exon 51 of the dystrophin gene would cause a frame shift that would mostly restore the protein’s functionality and mitigate the symptoms of dystrophy. There are existing methods by which this solution can be implemented, but we are interested in identifying small molecules that can bind selectively to specific exons in pre-mRNA molecules and alter their three-dimensional conformation; and thus their splicing pattern. Since splicing depends on exon definition, i.e., the recognition of exons by spliceosomes, having a small molecule bind to the splicing activator of the unwanted exon would prevent that exon from being spliced into the final mRNA molecule.
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