BIOL 3134 Lecture Notes - Lecture 18: Aquadvantage Salmon, Cytomegalovirus Retinitis, Exon Skipping
Document Summary
Human genetics in clinical settings: genetic testing, genetic counseling, gene therapy, possibility for designer"s babies. Inserting a fluorescent protein into the dog"s genome: where in the genome the gene is inserted is essential. Various strategies: transgenic (add/increase, gene silencing (decrease, genome editing (alter) Gene silencing: decreasing mrna levels in target cells, not changing genome sequence, still challenging to deliver to target cells. Antisense oligonucleotide (aso: treatment for cytomegalovirus retinitis in immunocompromised patients, withdrawn from market in 2006, represses a viral gene, required for viral replication, treatment for duchenne muscular dystrophy. Genome editing: changing genome sequence, most recent technology, crispr, cut and paste dna with ease and precision, guide rnas (address) and cas9 protein (scissors, a dna sequence of interest may be inserted or deleted. Genetically modified organisms (gmos: field tests may not adequately predict the effects on ecosystems, safety for human (especially long-term) is unknown. Somatic cells: corrects only the cells that a disease affects, not heritable.