Lecture

RNA-based Approaches to Disease Treatment

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In course

BIOENG-320: Synthetic biology

This advanced Bachelor/Master level course will cover fundamentals and approaches at the interface of biology, chemistry, engineering and computer science for diverse fields of synthetic biology. This class requires critical and analytical thinking at the ...

Description

This lecture covers the application of cutting-edge chemical and synthetic biology tools to discover and control actionable molecular targets for disease treatment, including engineered networks, drug-target identification, and therapeutic delivery. It explores the impact of synthetic biology on the therapeutic spectrum, focusing on biologics and small molecules. The lecture also delves into RNA-based approved drugs, programmable RNA-targeting tools, and case examples such as RNA interference pathways and CRISPR-Cas9 genome editing. Additionally, it discusses the development of RNA-based SARS-CoV-2 vaccine candidates and the methodology for programmable RNA targeting strategies.

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Instructors (3)

Vassily Hatzimanikatis

Dr. Vassily Hatzimanikatis is currently Associate Professor of Chemical Engineering and Bioengineering at Ecole Polytechnique Federale de Lausanne (EPFL), in Lausanne, Switzerland. Vassily received a PhD and an MS in Chemical Engineering from the California Institute of Technology, and his Diploma in Chemical Engineering from the University of Patras, in Greece. After the completion of his doctoral studies, he held a research group leader position at the Swiss Federal Institute of Technology in Zurich (ETHZ), Switzerland. Prior to joining EPFL, Vassily worked for three years in DuPont, Cargill, and Cargill Dow, and he has been assistant professor at Northwestern University, at Illinois, USA. Vassilys research interests are in the areas of computational systems biology, biotechnology, and complexity. He is associate editor of the journals Biotechnology & Bioengineering, Metabolic Engineering and Integrative Biology, and he serves on the editorial advisory board of the journals Bioprocess and Biosystems Engineering, Journal of Chemical Technology and Biotechnology, and Industrial Biotechnology. He has written over 70 technical publications and he is co-inventor in three patents and patent applications. Vassily is a fellow of the American Institute for Medical and Biological Engineering (2010), he was a DuPont Young Professor (2001-2004), and he has also received the Jay Bailey Young Investigator Award in Metabolic Engineering (2000), and the ACS Elmar Gaden Award (2011).

Patrick Daniel Barth

Professor Patrick Barth is Associate Professor at EPFL and Adjunct Associate Professor at Baylor College of Medicine, Houston, TX, USA. He received training in Physics, Chemistry and Biology (University of Paris, ENS) in France and performed his PhD at the Commissiariat a l'Energie Atomique in Saclay, France on structure/function studies of membrane proteins (photosystem I) using biochemical and biophysical experimental techniques. He carried out postdoctoral studies at University of California at Berkeley with Tom Alber on computational development for calculating protein electrostatics and designing de novo selective peptide inhibitors of cellular protein interactions. He then went to the University of Washington as a postdoctoral fellow and instructor in David Baker's laboratory to develop computational techniques in the software Rosetta for predicting and designing membrane protein structures. He started his independent career and received tenure at Baylor College of Medicine. He will continue at EPFL to marry computation and experiment for understanding the molecular determinants of signal transduction, as well as modeling and designing membrane proteins with novel functions for various synthetic biology and therapeutic applications.

Official source

Ontological neighbourhood

Biology

Genetics: Molecular genetics

Related concepts (27)

RNA

Ribonucleic acid (RNA) is a polymeric molecule that is essential for most biological functions, either by performing the function itself (Non-coding RNA) or by forming a template for production of proteins (messenger RNA). RNA and deoxyribonucleic acid (DNA) are nucleic acids. The nucleic acids constitute one of the four major macromolecules essential for all known forms of life. RNA is assembled as a chain of nucleotides.

RNA-dependent RNA polymerase

RNA-dependent RNA polymerase (RdRp) or RNA replicase is an enzyme that catalyzes the replication of RNA from an RNA template. Specifically, it catalyzes synthesis of the RNA strand complementary to a given RNA template. This is in contrast to typical DNA-dependent RNA polymerases, which all organisms use to catalyze the transcription of RNA from a DNA template. RdRp is an essential protein encoded in the genomes of most RNA-containing viruses with no DNA stage including SARS-CoV-2.

Transfer RNA

Transfer RNA (abbreviated tRNA and formerly referred to as sRNA, for soluble RNA) is an adaptor molecule composed of RNA, typically 76 to 90 nucleotides in length (in eukaryotes), that serves as the physical link between the mRNA and the amino acid sequence of proteins. Transfer RNA (tRNA) does this by carrying an amino acid to the protein synthesizing machinery of a cell called the ribosome. Complementation of a 3-nucleotide codon in a messenger RNA (mRNA) by a 3-nucleotide anticodon of the tRNA results in protein synthesis based on the mRNA code.

Antisense RNA

Antisense RNA (asRNA), also referred to as antisense transcript, natural antisense transcript (NAT) or antisense oligonucleotide, is a single stranded RNA that is complementary to a protein coding messenger RNA (mRNA) with which it hybridizes, and thereby blocks its translation into protein. The asRNAs (which occur naturally) have been found in both prokaryotes and eukaryotes, and can be classified into short (200 nucleotides) non-coding RNAs (ncRNAs).

Small molecule

In molecular biology and pharmacology, a small molecule or micromolecule is a low molecular weight (≤ 1000 daltons) organic compound that may regulate a biological process, with a size on the order of 1 nm. Many drugs are small molecules; the terms are equivalent in the literature. Larger structures such as nucleic acids and proteins, and many polysaccharides are not small molecules, although their constituent monomers (ribo- or deoxyribonucleotides, amino acids, and monosaccharides, respectively) are often considered small molecules.

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Related concepts (27)

RNA

RNA-dependent RNA polymerase

Transfer RNA

Antisense RNA

Small molecule