GeneticMedicines I-Feb3(s) Flashcards
(20 cards)
What are the three categories of genetic medicines?
- Somatic stem cells (SSCs)
- Gene transfer
- RNA modification
What is the difference between non-viral and viral transfection?
Non-viral transfection uses physical methods like electroporation and gene guns, while viral transfection uses viral vectors to deliver genetic material.
What are the different types of viral vectors used for gene transfer therapy?
- Adenovirus
- AAV (Adeno-Associated Virus)
- Retrovirus
Define RNA modification therapy.
RNA modification therapy involves targeting mRNA to suppress its levels or correct/add function.
How common are monogenic disorders?
They account for >10,000 heritable single gene disorders and occur in 10 out of 1000 births.
What is the primary goal of genetic medicines for monogenic disorders?
To repair or replace the mutated or missing gene/product and alleviate the unwanted phenotype.
What is metabolic manipulation?
The use of dietary modification and/or small molecule therapy to compensate for a deranged biological process.
What is protein augmentation?
The process of purifying the missing protein and administering it to the patient, particularly effective for extracellular protein deficiencies.
What does somatic stem cell therapy involve?
Introducing healthy stem cells into the affected organ or tissue.
What types of conditions can hematopoietic stem cell transplantation treat?
Lysosomal storage disorders, immunodeficiencies, hemoglobinopathies, and leukodystrophies.
What is essential for a successful stem cell transplantation?
The donor must have a matching HLA (human leukocyte antigen) system.
What is ex vivo gene transfer?
It is limited to disorders where the relevant cell population can be removed, genetically modified, and then replaced.
What is a cDNA in gene transfer therapy?
A complementary DNA sequence flanked by an active promoter and a transcription stop and polyadenylation site.
True or False: Viral vectors can permanently integrate into the host genome.
False. Some viral vectors can provide transient expression.
What type of cells can be targeted by retroviral vectors?
Dividing cells.
What are antisense oligonucleotides used for?
To decrease the levels of a mutant protein to favorably alter the phenotype in genetic diseases.
What is RNA interference (RNAi)?
A cellular pathway where dsRNA forms complexes with ribonucleases to silence specific mRNA.
What is the role of spliceosomes in RNA modification therapy?
They modify pre-mRNA by joining independent pre-mRNAs through trans-splicing.
How can large genes be addressed in RNA modification therapy?
By using two separate gene transfer vectors that are joined through spliceosome-mediated trans-splicing.
What components are included in a trans-splicing ribozyme?
A complementary hybridization domain, a nucleolytic motif, and a portion of the target mRNA.
