BIOL SCI 150 Lecture Notes - Lecture 19: Severe Combined Immunodeficiency, Adenosine Deaminase, Adaptive Immune System

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Getting dna into cells (cid:840)3 systems used to deliver genes for therapy(cid:841) Most humans are immune to adenovirus (cid:840)dna gets cleared(cid:841) Integrates into host genome using reverse transcriptase. Can cause mutations (cid:840)integration sites can be at bad places when treating large number of cells(cid:841) Can infect any cell (cid:840)non-dividing cells and dividing(cid:841) Adenovirus they wanted to deliver gene to liver. Unfortunately, bc adenovirus cause massive infection and triggers massive immune response leading to lethality. Now used in tissues where there isn"t an immune response. Most effective in eye and retina to treat genetic diseases of the retina. Treatment of progressive blindness (cid:840)adenovirus brought in a function copy of the rpe65 gene that was mutant in the patients(cid:841) Retrovirus the most famous is hi(; rna viruses that have ability to copy rna to dna to insert in host genome. Retroviruses carrying the normal adenosine deaminase gene were used to treat children with this.

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