siRNA and Molecular Therapies

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Questions and Answers

How many base pairs are typically found in a siRNA molecule?

  • 10
  • 50
  • 30
  • 20 (correct)

What protein is responsible for binding to one strand of siRNA in the RNA-induced silencing complex (RISC)?

  • Ago2 (correct)
  • RISC1
  • RISC2
  • Ago1

What is the primary outcome of siRNA silencing a gene?

  • The prevention of protein production (correct)
  • The production of a mutated protein
  • The degradation of the DNA sequence for the target gene
  • The increase in protein production

What is the major advantage of siRNA technology for drug development?

<p>It targets specific genes, allowing for highly targeted therapies (B)</p> Signup and view all the answers

What is the role of the RISC complex in siRNA-mediated gene silencing?

<p>Binding to and degrading target mRNA molecules (C)</p> Signup and view all the answers

What are some of the target tissues for siRNA-based therapies?

<p>Eyes, lungs, liver, skin, kidney and tumours (A)</p> Signup and view all the answers

What is a significant challenge of siRNA technology in drug development?

<p>The potential for off-target effects and immune responses (A)</p> Signup and view all the answers

Who were the scientists awarded the Nobel Prize for their discovery of the process of siRNA?

<p>Andrew Fire and Craig Mello (C)</p> Signup and view all the answers

What are the two main drug targets discussed in the content?

<p>Receptors and Enzymes (A)</p> Signup and view all the answers

Which technology is NOT discussed in the content?

<p>CRISPR (D)</p> Signup and view all the answers

What is the primary goal of this presentation?

<p>To introduce various molecule-based therapies and their market status (C)</p> Signup and view all the answers

What is the purpose of the 'Market Story' sections in the presentation?

<p>To provide an overview of the market potential and current state of each technology (A)</p> Signup and view all the answers

Which of the presented technologies is described as a defense against viruses?

<p>siRNA (C)</p> Signup and view all the answers

What is the primary function of blocking peptides, as described in the content?

<p>To disrupt the interaction between proteins by inhibiting their binding (A)</p> Signup and view all the answers

What is the main purpose of discussing receptor trafficking and anchoring proteins?

<p>To show how these proteins can be targeted by blocking peptides (B)</p> Signup and view all the answers

How do blocking peptides differ from aptamers in their mechanism of action?

<p>Blocking peptides are synthesized chemically while aptamers are selected by evolution (D)</p> Signup and view all the answers

What is the key difference between siRNA's and aptamers' function?

<p>siRNA targets specific genes while aptamers inhibit protein interactions (B)</p> Signup and view all the answers

How do aptamers contribute to toxin removal?

<p>By binding to and neutralizing toxins, preventing them from interacting with target cells (B)</p> Signup and view all the answers

What is the essential role of transport proteins in controlling receptor expression?

<p>They transport receptors to the cell surface for activation (A)</p> Signup and view all the answers

Why is the postsynaptic density considered to be full of proteins?

<p>Because it's the site of neurotransmitter release and signal transduction (B)</p> Signup and view all the answers

How can blocking peptides be used as receptor activators?

<p>By blocking the binding site of an inhibitory ligand (B)</p> Signup and view all the answers

Why is the creation of blocking peptides considered a novel method to alter receptor function?

<p>They offer a targeted approach to modulate receptor activity, unlike traditional drugs (D)</p> Signup and view all the answers

What key benefit of blocking peptides is emphasized in the content?

<p>They are highly specific and target only specific receptors (D)</p> Signup and view all the answers

Which statement best describes the current market story for blocking peptides?

<p>Blocking peptides are still under development and research, with only a few candidates in clinical trials (D)</p> Signup and view all the answers

Which of the following drug types are NOT mentioned as major categories currently on the market?

<p>Antibody inhibitors (D)</p> Signup and view all the answers

What is the first biologic drug mentioned in the text?

<p>Humanised insulin (D)</p> Signup and view all the answers

Which protein is not a target of a biologic drug listed in the table?

<p>EGF (B)</p> Signup and view all the answers

According to the table, which drug is the most expensive?

<p>Comirnaty (B)</p> Signup and view all the answers

Which of the following is not a type of biologic drug mentioned in the text?

<p>Small molecules (D)</p> Signup and view all the answers

How does siRNA technology directly target the production of proteins?

<p>By degrading mRNA molecules that code for the targeted protein (D)</p> Signup and view all the answers

What is the primary function of DICER in the antiviral defense system?

<p>It produces small interfering RNA (siRNA) from viral RNA (A)</p> Signup and view all the answers

What is the ultimate outcome of the interaction between siRNA/RISC complex and mRNA?

<p>The prevention of protein production (C)</p> Signup and view all the answers

What is the main purpose of siRNA technology?

<p>To inhibit the production of specific proteins (D)</p> Signup and view all the answers

What is the role of mRNA in protein synthesis?

<p>It carries the genetic code from DNA to the ribosomes where proteins are made (A)</p> Signup and view all the answers

Which of the following describes the process of translation in protein synthesis?

<p>The production of a protein based on the genetic code in mRNA (C)</p> Signup and view all the answers

What is the role of post-translational modifications in protein synthesis?

<p>They add or remove chemical groups to a protein, affecting its function (B)</p> Signup and view all the answers

What is the primary goal of using siRNA technology in therapeutic applications?

<p>To prevent the expression of disease-causing genes or proteins (C)</p> Signup and view all the answers

How does the siRNA/RISC complex contribute to gene silencing?

<p>It degrades the mRNA transcripts that code for the targeted protein (B)</p> Signup and view all the answers

Which of the following is a potential advantage of using siRNA technology as a therapeutic strategy?

<p>It can specifically target and silence disease-related genes (C)</p> Signup and view all the answers

Flashcards

siRNA

Small interfering RNA used for gene silencing.

Gene silencing

The process of preventing gene expression or silencing a gene.

RISC

RNA-induced silencing complex that binds siRNA and mRNA.

Ago2 protein

A component of RISC that helps bind siRNA.

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mRNA

Messenger RNA that carries genetic information for protein synthesis.

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Nobel Prize in 2006

Awarded to Andrew Fire and Craig Mello for discovering siRNA.

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Therapeutic areas of siRNA

Tissue types and conditions targeted by siRNA therapy.

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Commercial siRNA synthesis

Service offered by companies to create siRNA sequences.

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Molecular Drug Targets

Components like DNA, RNA, and proteins targeted by drugs.

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Biologics

Biological products such as antibodies and proteins used as therapies.

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First Biologic Drug

Humanized insulin, introduced in 1982.

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Approval of Biologics

Increase in biologics approval, from 6 in 2010 to 15 in 2022.

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Top Selling Drugs 2021

In 2021, 12 of the top 20 drugs were biologics.

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Transcription

Process of converting DNA to mRNA.

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Translation

Process of synthesizing proteins from mRNA.

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RISC Complex

RNA-induced silencing complex that uses siRNA to silence mRNA.

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Antiviral Defense

Cellular response involving siRNA to combat viral RNA.

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DICER

Enzyme that cuts viral RNA into smaller fragments.

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Post-translational Modification

Changes made to proteins after translation that affect function.

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Antibody

A protein used by the immune system to identify and neutralize pathogens.

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Receptor Agonists

Drugs that activate receptors to produce a biological response.

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Molecular-based Therapies

Medical treatments that utilize molecular mechanisms, including siRNA, aptamers, and peptides.

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Mechanism of Action

The specific biochemical interaction through which a drug produces its effect.

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Drug Targets

Molecules, typically proteins, that drugs interact with to produce a therapeutic effect.

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Receptors

Protein molecules on cell surfaces that receive signals from drugs and hormones.

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Aptamers

Short, single-stranded RNA or DNA that binds specifically to target molecules.

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Blocking Peptides

Short proteins that inhibit or activate receptor function.

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Transport Proteins

Proteins that move substances across cell membranes.

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Drug Labelling

Using aptamers to tag drugs for targeting and tracking delivery.

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Market Activity

Analysis of the commercial landscape for specific drug technologies.

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Benefits and Challenges

Pros and cons associated with the use of a therapy or technology.

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Toxin Removal

Using aptamers to bind and remove harmful substances from the body.

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Receptor Trafficking

The process of moving receptors to and from the cell surface.

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Study Notes

Molecular-Based Therapies and Technologies: siRNA

  • siRNA (small interfering RNA) is a molecular-based technology used to inhibit protein production by targeting mRNA for destruction.
  • The process mirrors the body's antiviral defense. Viral RNA is cut into smaller pieces (siRNA) by DICER, which then bind to RISC (RNA-induced silencing complex). RISC binds to one strand of siRNA, and the complex targets corresponding mRNA. The targeted mRNA is cut, silencing the gene.
  • To use siRNA therapeutically, a specific siRNA sequence matching the target mRNA needs to be created. This is synthesized, then introduced to cells.
  • RISC binds to one strand and the siRNA/RISC complex binds the target mRNA, cutting it, and silencing the gene.
  • siRNA has been trialled in various tissues (eyes, lungs, liver, skin, kidney, tumors).Therapeutic areas explored include age-related macular degeneration (AMD), asthma, metabolic diseases, dermatology, renal failure, and cancers.

Drug Targets and Biologics

  • Major drug targets are receptors and enzymes, but also DNA, mRNA, and proteins.
  • Traditional drugs include receptor agonists, antagonists, and enzyme inhibitors (small molecules).
  • Biologics (e.g., antibodies, proteins, peptides, DNA/RNA molecules, synthetic vaccines) are increasingly important. More biologics were approved in 2022 than in 2010.
  • Biologics, including antibodies and protein-based drugs, dominate the top 20 selling drugs (2021).
  • Humanized insulin was the first biologic drug (1982).

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