m Flashcards
What are biopharmaceuticals?
Biopharmaceuticals can be:
* Proteins
* Nucleic acids
* Cells or tissues
Biopharmaceuticals are produced using a living system.
What is the molecular weight of Aspirin?
180 Da
What is the molecular weight of a monoclonal antibody?
150,000 Da
What is insulin?
51-amino acid protein, synthesized as a proinsulin precursor in the β-cells of the pancreas
What are the two polypeptide chains in insulin?
A-chain and B-chain
How many amino acids are in the A-chain of insulin?
21 amino acids
How many amino acids are in the B-chain of insulin?
30 amino acids
What type of bonds connect the polypeptide chains in insulin?
Disulfide bonds
What year was insulin discovered?
1921
What year was the first successful use of animal insulin in humans?
1922
What year did the FDA approve recombinant human insulin (Humulin)?
1982
What are the benefits of recombinant insulin?
- Reduced risk of allergic reactions
- Better glucose control
- Mass production meets global demand
- Engineered for specific patient needs
What is the onset time for rapid-acting insulin?
5 - 15 min
What is the peak time for short-acting insulin?
2 - 3 hr
What is the duration of long-acting insulin?
20 - 24 hr
What are interferons?
A group of signaling proteins produced by eukaryotic cells in response to pathogens, tumors, and other biological inducers
What are the biological roles of interferons?
- Antiviral
- Immunomodulatory
- Antiproliferative
What are the three types of interferons?
- Type I: IFN-α, IFN-β
- Type II: IFN-γ
- Type III: IFN-λ
What are some medical applications of interferons?
- Treatment of viral infections (e.g., hepatitis B and C)
- Cancer therapy (e.g., melanoma, leukemia)
- Multiple sclerosis
What is PEGylation?
A process that covalently attaches a polyethylene glycol (PEG) polymer to a macromolecule
What is a monoclonal antibody?
Laboratory-produced molecules engineered to serve as substitute antibodies
What year was the first FDA-approved monoclonal antibody?
1986
What are the types of monoclonal antibodies based on their origin?
- Murine
- Chimeric
- Humanized
- Fully Human
What is the challenge associated with murine monoclonal antibodies?
High immunogenicity
What is the benefit of humanized monoclonal antibodies?
Improved efficacy and reduced risk of immune reactions
What is CAR T-cell therapy?
A type of immunotherapy that uses genetically modified T cells to target and destroy cancer cells
What are the steps in CAR T-cell therapy?
- T cell collection
- Genetic modification
- Expansion
- Infusion
What are the two key properties of stem cells?
- Self-renewal
- Differentiation
What are the types of stem cells?
- Embryonic Stem Cells (ESCs)
- Adult Stem Cells
- Induced Pluripotent Stem Cells (iPSCs)
What are the advantages of stem cell therapy?
- Regenerative potential
- Minimally invasive
- Reduced risk of rejection
- Potential to cure previously untreatable diseases
What is an antisense oligonucleotide?
Short, synthetic single strands of nucleic acids designed to bind to specific RNA molecules to modulate gene expression
What are some clinical uses of antisense oligonucleotides?
- Treatment of genetic disorders
- Cancers
- Viral infections
What are antisense oligonucleotides (ASOs)?
Short, synthetic single strands of nucleic acids designed to bind to specific RNA molecules to modulate gene expression.
What are the clinical uses of antisense oligonucleotides?
Treatment of genetic disorders, cancers, and viral infections. Examples include:
* Spinal Muscular Atrophy (SMA)
* Duchenne Muscular Dystrophy (DMD)
* Homozygous familial hypercholesterolemia (HoFH)
* Cytomegalovirus retinitis
* Hereditary transthyretin amyloidosis.
How do antisense oligonucleotides modulate RNA function?
Through:
* RNA degradation: RNase H-mediated cleavage of target RNA
* Splicing modulation: Correcting aberrant splicing
* Translation blockade: Preventing ribosome binding.
What are the advantages of using antisense oligonucleotides?
Advantages include:
* Highly specific targeting of disease-causing RNA
* Can be designed for a wide range of genetic mutations
* Long-lasting effects with periodic dosing
* Does not alter the genome, reducing risks of insertional mutagenesis.
Name two clinical applications of antisense oligonucleotides.
Examples include:
* Mipomersen for Homozygous familial hypercholesterolemia (HoFH)
* Nusinersen (Spinraza) for Spinal Muscular Atrophy (SMA).
What is gene therapy?
A medical approach that treats or prevents disease by correcting the underlying genetic problem by introducing a functional gene into cells.
What is the procedure for gene therapy?
Steps include:
* Identify the defective gene causing the disease
* Develop a healthy copy of the gene
* Deliver the healthy gene into the patient’s cells using a vector
* The healthy gene integrates into the cell’s DNA.
What are the two types of gene therapy?
Types include:
* Somatic Gene Therapy: Targets non-reproductive cells
* Germline Gene Therapy: Targets reproductive cells.
What is a key difference between somatic and germline gene therapy?
Somatic Gene Therapy does not pass changes to future generations, while Germline Gene Therapy does.
What are viral vectors in gene therapy?
Modified viruses used to deliver genes into cells, which are efficient but can trigger immune responses.
What are non-viral methods of gene delivery?
Methods include:
* Nanoparticles
* Liposomes
* Direct DNA injection.
What are the advantages of viral vectors?
High transfection efficiency.
What are the disadvantages of viral vectors?
Disadvantages include:
* High immunogenicity
* High production cost
* Low packaging capacity.
What is a key advantage of non-viral gene delivery methods?
Better safety profile.
What is a key disadvantage of non-viral gene delivery methods?
Lower transfection efficiency.
List three applications of gene therapy.
Applications include:
* Genetic Disorders
* Cancer
* Infectious Diseases.
What are two FDA-approved gene therapies?
Examples include:
* Luxturna: inherited retinal disease
* Zolgensma: spinal muscular atrophy.
