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Time: 8AM PST | 11AM EST | 5PM CET
Date: February 22, 2024
Title: Advancing Therapeutic Approaches for Erythropoietic Protoporphyria
Description: Currently, no curative treatment is available for the rare disease erythropoietic protoporphyria (EPP). The genetic background of EPP is well understood; a loss-of-function mutation on one ferrochelatase (FECH) allele in trans to a single nucleotide polymorphism (SNP) on the other leads to mis-splicing and degradation of the FECH mRNA. Deficiency of the FECH enzyme in the haem biosynthesis pathway leads to the accumulation of the phototoxic metabolite protoporphyrin IX (PPIX). Upon exposure to sunlight, EPP patients experience acute severe pain and burn-like injuries, and some may develop hepatic complications. In the Hall lab, a mouse model of EPP has been developed. This model allows us to test therapeutic approaches for the disease, such as small molecules, splice-switching antisense oligonucleotides, and CRISPR-based gene editing.
Speaker:
Title: Self Assembled Nanoparticles for Delivering Functional Nucleic Acids
Description: Nucleic acids have been synthesized chemically for decades using high yielding phosphoramidite chemistry and solid phase synthesis, to controllably grow sequence-defined oligonucleotides. The same chemistry can be used to incorporate unnatural monomers that can self-assemble into nanoparticles. These nanoparticles have been applied to the delivery of functional nucleic acids, including antisense oligonucleotides. By exploring minute changes in their structure, we can observe differences in their interactions with serum protein and their effectiveness at delivering nucleic acid payloads.
