Gene therapy is a medical approach that involves introducing genetic material into cells to treat or prevent a disease.
What is the goal of gene therapy?
The goal of gene therapy is to replace, modify, or supplement a faulty or missing gene to restore normal function.
How is genetic material delivered into target cells in gene therapy?
Genetic material is delivered into target cells using various delivery methods.
What are the main goals of gene therapy?
- Treat genetic disorders by replacing or repairing defective genes.
- Treat acquired diseases like cancer and heart disease.
- Prevent disease development before symptoms appear.
- Enhance desired traits, though this is controversial.
What is one potential application of gene therapy for acquired diseases?
Gene therapy can introduce genetic material to treat acquired diseases like cancer, heart disease, and neurodegenerative disorders.
How can gene therapy prevent disease?
Gene therapy can introduce genetic material to prevent the development of a disease before symptoms appear.
What is gene addition in gene therapy?
Gene addition involves introducing a normal, functional copy of a gene to compensate for a defective one.
What is gene editing in gene therapy?
Gene editing uses tools like CRISPR to directly modify or repair a defective gene.
What is gene silencing in gene therapy?
Gene silencing involves blocking or reducing the expression of a problematic gene.
What are the potential benefits of gene therapy?
- Treat genetic disorders by addressing the root cause.
- Treat acquired diseases like cancer and heart disease.
- Prevent disease development before symptoms appear.
- Provide a long-term or permanent solution.
What are the potential risks of gene therapy?
- Safety concerns: immune reactions, insertional mutagenesis, off-target effects.
- Delivery challenges: difficulty in delivering genetic material to target cells.
- Ethical considerations: concerns around genetic enhancement.
- Cost and accessibility: high costs limiting availability.
Mechanisms of gene therapy Genetic screening
DNA sequencing
Gene delivery
genetic screening:
Scanning of an individuals genetic makeup to find heritable defects that causes the genetic disorders or may cause disorders in the near future
Methods of genetic screening
Molecular genetic tests/gene test
Chromosomal genetic tests
Biochemical genetic tests
DNA sequencing is the process of determining the order of nucleotides in a DNA molecule
What is the primary use of DNA sequencing?
To determine the sequence of individual genes, larger genetic regions, full chromosomes, or entire genomes.
How does DNA sequencing contribute to biological research?
It provides the order of individual nucleotides in DNA or RNA from various sources.
In which fields has knowledge of DNA sequences become indispensable?
Basic biological research, medical diagnosis, biotechnology, forensic biology, virology, and biological systematics.
What can researchers identify using information obtained from DNA sequencing?
Changes in genes, associations with diseases and phenotypes, and potential drug targets.
Why is DNA sequencing significant in evolutionary biology?
It helps study how different organisms are related and how they evolved.
What is metagenomics?
It involves the identification of organisms present in various environments.
Why is knowing which organisms are present in an environment critical?
It is essential for research in ecology, epidemiology, microbiology, and other fields.
How does DNA sequencing aid in understanding microbiomes?
It enables researchers to determine which types of microbes may be present in a microbiome.
How can medical technicians use DNA sequencing?
They may sequence genes or complete genomes of patients to assess the risk of genetic diseases.
What is genetic testing?
It is the process of analyzing genes to determine the risk of genetic diseases.
How is DNA sequencing used in forensic identification?
It may be used along with DNA profiling methods for forensic identification and paternity testing.
What types of samples can DNA sequencing be performed on?
Samples from animals, plants, bacteria, archaea, or virtually any other source of genetic information.
What does DNA sequencing reveal about proteins?
It helps study genomes and the proteins they encode.
How does DNA sequencing contribute to the field of virology?
It provides insights into viral genomes and their interactions with hosts.
What is the relationship between DNA sequencing and ecological research?
DNAsequencing helps identify organisms in various environments, aiding ecological studies.
What is the purpose of paternity testing?
To determine biological relationships between individuals
Who first described the sequencing of nucleotides in DNA?
Allen Maxam and Walter Gilbert
What method did Frederick Sangar develop for DNA sequencing?
The dideoxy chain termination method
What are ddNTPs in the context of DNA sequencing?
Dideoxyribonucleoside triphosphates
What happens when a ddNTP is incorporated into a growing DNA strand?
It terminates the growing DNA strand
Why does the incorporation of a ddNTP terminate the DNA strand?
Because it lacks a 3'-OH group
What distinguishes each ddNTP in the sequencing process?
Each ddNTP is tagged with a distinct fluorescent marker
What is the advantage of third generation sequencing compared to earlier methods?
It is much faster and lessexpensive
What is the shotgun sequencing method used for?
Sequencing DNA segments longer than 1000 bp or whole genomes
How does the shotgun sequencing method work?
Target DNA is broken into random fragments, sequenced independently, and reassembled