SCIE 1P51 Lecture Notes - Lecture 11: Up To Here, Lesko, Down Syndrome
16 Sep 2016
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Not producing the proper long lived red blood cells no oxygen within. Trying to reverse the process; getting rid of the certain gene sequence (have identified it [where its located]) Limited success in being able to shut down the bad proteins. Mice have only lived 2-3 months in the lab. Dominant heterozygous (good copy & bad copy) No idea that you have it (certain gene sequence) not getting signals to various parts of your body. Repeated bases of cag in the dna sequence. Dna screen (if you know its within the family); screen future offspring to see if they are carrying that particular gene sequence. Chromosome 4 editing the geno = gene therapy. ~1 in 5000 live male births are affected. Over 60% of persons with haemophilia treated with plasma concentrates in the early 1980s became hiv+ Phenotype indistinguishable from haemophilia a severity related to factor ix level.
