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Recombinant 2
Immunotherapy
| Question | Answer |
|---|---|
| What is gene therapy? | The use of nucleic acids as therapeutic medicinal compounds |
| State the 3 strategies used with gene therapy | Replacing a mutated gene that caused disease with a healthy copy of the gene Inactivating or knocking out a mutated gene that isn't functioning properly Introducing a new gene into the body to help fight a disease |
| Explain in vivo gene therapy | Virus carries the healthy gene, the healthy gene gets into cells, starts making healthy proteins. Can be injected intratumour, intravenous, intramuscular, intraperitoneal, intra-arterial. |
| Explain ex vivo gene therapy | Healthy genes are inserted into donor cells, cells start making healthy proteins, are readministered via a catheter. |
| List the four ways that gene therapy is used in clinical settings | Therapy for cancers - correction of genetic mutations Therapy for vascular disease Therapy for monogenetic inherited disorders Therapy for infectious disease |
| What is immunotherapy? | A type of cancer treatment that helps your immune system fight cancer. |
| What is a Car-T therapy? | Chimeric Antigen Receptor T Cell. Combines the recognition specificity of a MAB with T Cell function to kill cancer cells. |
| What are the draw backs to Car-T therapy? | Serious side effects and the significant cost |
| How do Car T therapies work? | The Car T cells bind to the tumour antigen protein on the surface of the cancer cell. Downstream signalling activates the T cell's 3 functions: immune cell activation, cytotoxic activity and proliferation. Causes cancer cell apoptosis + tumour regression. |
| How are Car T cells made? | T cells are extracted from the patients blood (leukapheresis) T cells are stimulated into cytotoxic T cells (CD8's) Cell engineering (autologous vs allogeneic) Cell proliferation (create army of T cells) Cell infusion (infused into patients) |
| What is dendritic cell activated cytokine induced killer cell mediated immunotherapy? | An ex vivo culture without genetically modified cells |
| What is a viral vector for gene transfer? | Viruses, natural parasites that efficiently enter target cells, take over processes for propagation. Most effective vectors for gene therapy. 70% of gene therapy clinical trials use viral vectors. |
| Why do saefty concerns exist for use of viral vectors? | Viral vectors are still foreign material, so the pathogenic and infective bits need to be removed. Possibility of infection if not produced to standard. |
| List the viruses used as viral vectors for gene therapy | Retrovirus, lentivirus, adenovirus, adeno-associated viral vectors, poxvirus, herpes simplex virus |
| How do non-viral vectors for gene therapy compare? | Are double-stranded recombinant DNA plasmids, or encapsulated cationic polymer/lipid based formulations. Less immunogenic, so don't stimulate the immune system as much. Can be given multiple times without inhibiting immune response.Cheap to produce. |
| How do non-viral vectors for gene therapy compare cont'd? | Produce less un-wanted by products compared to viral. But: low transduction effiency, poor delivery to therapeutic target and cannot achieve sustained gene expression |
| Why do we use vaccines? | To protect humans and animals from infectious diseases |
| What is the principle of vaccination? | Mimicking an infection in a way that the immune system is activated when it encounters a pathogen, but the host will not suffer the normal results of a natural infection. |
| What are the characteristics of an ideal vaccine? | 100% efficient in all individuals of any age Provides lifelong protection after single administration Does not evoke any adverse reaction Is stable under various conditions Is easy to administer, perferably orally Is cheap to produce |
| What is the first step in criteria for vaccine formulation? | Selection of vaccine immunogen: Pick the right immunogens that are likely to induce immune response or must be the appropriate target and induce immune responses that are effective against the pathogen. |
| What is the second step in design criteria for vaccine formulation? | Adjuvants and delivery systems: magnifies the immune response to the vaccine Stimulate the immune system by 3 mechanisms: depot effect leading to slow antigen release, prolonging exposure Attraction and stimulation of APC's, delivery to lymp nodes |
| What is the third step in design criteria for vaccine formulation? | Determine immunisation schedule Depends on the type, magnitude, duration of the immune response. Involves determining initial immunisation and any subsequent boosters |
| What is the fourth step in design criteria for vaccine formulation? | Determining route of administration Parenteral delivery is common. Oral delivery can also be used. |
| What is the fifth step in design criteria for vaccine formulation? | Stability: Formulation needs to avoid degradation, liquid formulation vs powder for reconstitution Temperature control, which if poor can degrade the antigen, impacting safety and effectiveness of the vaccine |
Created by:
LDM
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