MCB 121 Lecture 17

MCB 121 Lecture 17 - MCB 121 Lecture 17 I Gene Therapy II...

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MCB 121 Lecture 17 I. Gene Therapy II. Engineered Livestock <= I. and II. are still experimental III. Engineered Plants. ------------------------------------ Gene Therapy: Purpose: introduce genes with therapeutic effect Difficulty: have to target the correct cells of the organisms Simplest system: self-renewing system. If the cell is self-renewing, we are able to change the whole system. Early target: Blood Diseases => get to blood stem cells to replace the blood over time with the cells you genetically altered. => Once the cells are genetically altered, they need to be introduced Method of introducing: 1) Free DNA deliver: Problem: low efficiency and requires selection of results On the culture dish, cells have the tendency to over-grow => cells move toward cancer Selection allows the cells to have greater chance to become tumor cell 2) Viral vector - Infect cells efficiently - most commonly used ones a) Retrovirus: - has RNA genome - convert RNA into DNA inside the cell by reverse transcriptase - then insert the DNA into chromosome of the host - most commonly use retrovirus: => Lentivirus : - can infect non-dividing cells - example of lentivirus such as HIV (remove all the disease causing genes from HIV and put in the gene of interest) - the virus are usually "disabled": remove function necessary for virus to replicate. => Adenovirus : - double stranded DNA self-replicating virus - do not insert - causes minor infection (can be made incompetent) - it's transient expression (infection would persist for a period of time and lost)
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Two things that people target: 1) Genetic disease 2) Cancer First Case: Apr 2000, in France - Disease: SCIDX-1 = no immune system (boy with bubble disease) - Patient is missing γc cytokine receptor, which is necessary for T-cell proliferation => don't even need to select since every T cells can proliferate with the inserted gene. - Treatment: Retroviral vector => took out blood stem cells from bone marrow of patient => did in vitro infection of blood stem cells and culture these cells => reinject those cells back into the patient - Result: => 10 months post treatment = patient has normal immune function - Did 9 more patient => 9/11 were "cured" => 2 of 9 had severe leukemia. - Why there was leukemia?
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MCB 121 Lecture 17 - MCB 121 Lecture 17 I Gene Therapy II...

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