BIMS 201 Lecture 8: Chapter 17A Notes

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Texas A&M University
Biomedical Science
BIMS 201
Elizabeth A.Crouch

41017 Chapter 17A Notes Wrongful birth bill Problem=can viral vector travel to germ cells Defining whats severe and not severe microRNAs (miRNA) = complimentary to mRNA o shuts off protein production o transcribed from opposite strand of DNA o makes doublestranded RNA sequence o 22ish base pairs long and controls production level of proteins o antisense RNAs o can be complementary to badgood protein have to be looked @ so they dont shut off good protein production when genes inserted for therapy o mRNA and DNA are always made 5 3 ALD: demyelinating disease of microglial cells (begin in bone marrow and travel to brain) o Progressive brain deterioration bc demyelinating neurons = NOT transmitting signals o Defect in pore protein (ABCD1) in peroxisomes Protein makes pore in peroxisome that allows enzyme in that breaks down long chain FAs wo protein: long chain FAs stay o Xlinked disease ( > boys) Can live normal life once treated o Successful gene therapy using HIVstripped of everything except outer cell (doesnt have genes that cause AIDS, has good genes for ABCD1 and viral code protein that targets it to bone marrowmicroglial cells) o Newborn screen being dev. for it Chimeric o Deliver altered gene to Thelper cells (CD4+ cells) using modified HIV virus o Can train cells to target specific cancer cells o Leads to immune response thats highly specific to your cancer
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