HMM101 Introduction to Medical Biotechnology

A collection of flashcards based on the key concepts from HMM101 lectures on nucleic acid therapeutics.

What are the types of nucleic acid therapeutics discussed in HMM101?

Antisense oligonucleotides (ASOs), RNA interference (RNAi), ribozymes, and aptamers.

What is the main advantage of nucleic acid therapeutics?

They can target a wider range of druggable targets than traditional pharmaceuticals.

What is the primary function of ribozymes?

Ribozymes act as catalytic enzymes to cut RNA.

What is the difference between base pairing and shape recognition?

Base pairing involves complementary nucleobases joining together, while shape recognition allows molecules to bind based on their three-dimensional structures.

What is antisense oligonucleotide (ASO) therapy?

ASO therapy employs short strands of nucleic acids that bind to mRNA to inhibit gene expression.

What is the role of siRNA in gene silencing?

siRNA regulates gene expression by degrading target mRNA.

What are the challenges in delivering RNA therapeutics?

Delivery challenges include degradation by nucleases, large molecular size preventing cellular uptake, and off-target effects.

What is the significance of the Human Genome Project?

It aids in the identification of genes associated with diseases and potential drug targets.

What is the main goal of personalized medicine in the context of therapeutics discussed in HMM101?

To tailor treatments based on individual genetic makeup and ensure more effective and targeted therapies.

What are aptamers, and how are they used as therapeutics?

Aptamers are oligonucleotides that can bind specific targets; they are used to modulate biological functions or serve as therapeutics.

What are the potential risks associated with gene therapy?

Risks include insertional mutagenesis, immune responses, and off-target effects.

What is the purpose of clinical trials in the context of nucleic acid therapeutics?

To assess the safety, efficacy, and optimal delivery methods for promising therapeutic technologies.

How do ASOs prevent translation of mRNA?

By binding to mRNA, ASOs can block interactions with ribosomes and/or cause degradation of mRNA.

What therapeutic applications are being explored for microRNA (miRNA)?

miRNA therapeutics are being explored for various disorders, including cancer, cardiovascular diseases, and neurological conditions.

Describe the role of personalized medicine in RNA-based therapies.

Personalized medicine in RNA therapies focuses on tailoring treatments based on an individual's genetic profile to enhance therapeutic efficacy.

What is the function of the RNA-induced silencing complex (RISC)?

RISC utilizes small RNA molecules to silence target mRNA by promoting degradation or inhibiting translation.

What ethical considerations are associated with RNA therapeutic research?

Ethical considerations include patient consent, potential misuse of genetic information, and long-term effects of genetic modifications.

Who were the pioneers in the development of RNA therapeutics?

Key figures include J. D. Fire and A. Mello, who demonstrated gene silencing with RNA interference.

What is the mechanism of action for microRNA (miRNA) therapeutics?

miRNA therapeutics regulate gene expression by inhibiting translation or promoting degradation of target mRNA.

What are the primary delivery methods for RNA-based therapeutics?

Methods include lipid nanoparticles, viral vectors, and direct injection.

How can RNA therapeutics be utilized in cancer treatment?

They can specifically target oncogenic mRNAs or RNA regulators to inhibit tumor growth.